Literatura académica sobre el tema "Disease-modifying treatments"

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Artículos de revistas sobre el tema "Disease-modifying treatments"

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Galimberti, Daniela y Elio Scarpini. "Disease-modifying treatments for Alzheimer’s disease". Therapeutic Advances in Neurological Disorders 4, n.º 4 (20 de abril de 2011): 203–16. http://dx.doi.org/10.1177/1756285611404470.

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Waubant, Emmanuelle, Gavin Giovannoni, Chris Hawkes y Michael Levy. "Vaccines and disease-modifying treatments". Multiple Sclerosis and Related Disorders 26 (noviembre de 2018): A1—A2. http://dx.doi.org/10.1016/j.msard.2018.10.017.

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Weilbach, Franz X. y Ralf Gold. "Disease Modifying Treatments for Multiple Sclerosis". CNS Drugs 11, n.º 2 (1999): 133–57. http://dx.doi.org/10.2165/00023210-199911020-00005.

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Cummings, Jeffrey L. "Integrating Symptomatic- and Disease-Modifying Treatments". CNS Spectrums 13, S16 (2008): 28–30. http://dx.doi.org/10.1017/s1092852900027024.

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Although treatments for Alzheimer’s disease (AD) currently focus on symptomatic therapies, we are entering into an era of disease-modifying therapies. Central to disease modification is early diagnosis; the disease should be slowed as early as possible, maximizing the preservation of cognitive integrity. Ideally, AD should be diagnosed before the onset of dementia, perhaps with the use of biomarkers.Some therapies suggest that cholinesterase inhibitors and memantine have disease-modifying properties, though not all studies agree. Doody and colleagues have produced data suggesting that these agents may modify the course of AD, but it is not clear that they affect the underlying mechanisms that lead to cell death. Rather than being disease-modifying agents, cholinesterase inhibitors and memantine have potential as disease-course-modifying agents. Language precision will be extremely important in describing these therapies. One European consensus conference concluded that affecting disease course is not adequate for disease modification, but this has not been largely endorsed.Symptomatic therapies are those affecting the course of the disease. Their benefits are multidimensional, improving cognition, global assessment, activities of daily living, behavior, and caregiver burden. Symptomatic therapies should defer decline. Clinical trials show that symptomatic therapies produce an initial improvement above baseline. However, some patients experience observable changes and some experience none. These therapies are believed to produce ~1 point improvement on the Mini-Mental State Examination (MMSE) average, and a decline that is otherwise parallel to a placebo group after a period of delayed progression (Slide 1).
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Thomas, Emily, Brenda Wasunna‐Smith y Tarun Kuruvilla. "Aducanumab and disease modifying treatments for Alzheimer's disease". Progress in Neurology and Psychiatry 25, n.º 3 (julio de 2021): 4–6. http://dx.doi.org/10.1002/pnp.711.

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Roberts, S., J. Hendrick, A. Vinitsky, D. Barten, D. Izzarelli, M. Lewis, B. Robertson et al. "Symptomatic and Disease Modifying Treatments of Alzheimer's Disease". CNS Drug Reviews 6 (7 de junio de 2006): 19. http://dx.doi.org/10.1111/j.1527-3458.2000.tb00168.x.

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Kieseier, Bernd C. y Heinz Wiendl. "Oral Disease-Modifying Treatments for Multiple Sclerosis". CNS Drugs 21, n.º 6 (2007): 483–502. http://dx.doi.org/10.2165/00023210-200721060-00005.

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Comi, Giancarlo. "Disease-modifying treatments for progressive multiple sclerosis". Multiple Sclerosis Journal 19, n.º 11 (23 de septiembre de 2013): 1428–36. http://dx.doi.org/10.1177/1352458513502572.

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The last 20 years have seen major progress in the treatment of relapsing–remitting multiple sclerosis (RRMS) using a variety of drugs targeting immune dysfunction. In contrast, all clinical trials of such agents in primary progressive multiple sclerosis (PPMS) have failed and there is limited evidence of their efficacy in secondary progressive disease. Evolving concepts of the complex interplay between inflammatory and neurodegenerative processes across the course of multiple sclerosis (MS) may explain this discrepancy. This paper will provide an up-to-date overview of the rationale and results of the published clinical trials that have sought to alter the trajectory of both primary and secondary MS, considering studies involving drugs with a primary immune target and also those aiming for neuroprotection. Future areas of study will be discussed, building on these results combined with the experience of treating RRMS and new concepts emerging from laboratory science and animal models.
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Stamelou, Maria y Adam L. Boxer. "Disease-Modifying Treatments for Progressive Supranuclear Palsy". Movement Disorders Clinical Practice 2, n.º 1 (2 de febrero de 2015): 3–5. http://dx.doi.org/10.1002/mdc3.12142.

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Cummings, Jeffrey L. "Defining and labeling disease-modifying treatments for Alzheimer's disease". Alzheimer's & Dementia 5, n.º 5 (septiembre de 2009): 406–18. http://dx.doi.org/10.1016/j.jalz.2008.12.003.

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Tesis sobre el tema "Disease-modifying treatments"

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Gafson, Arie R. "Predicting response to disease modifying treatment in multiple sclerosis". Thesis, Imperial College London, 2017. http://hdl.handle.net/10044/1/56612.

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Multiple sclerosis (MS) is an autoimmune disease affecting the central nervous system (CNS) that most commonly begins with a relapsing-remitting course (RRMS). Many disease modifying treatments now are available, but none have efficacy in all patients, all are expensive and all are associated with possible adverse events. Stratifying patients to the best tolerated and most efficacious treatment either prior to or soon after commencing treatment would enhance relative benefits and reduce harm. Effective stratification depends on an understanding of relevant aspects of a drug’s mechanism of action, characterisation of key pharmacodynamic effects and being able to monitor disease activity over time. In this study, I set out to determine whether multi-omics profiling (transcriptome, cytokines, lipoproteins and metabolome) can fulfil these three requirements for one of the newer, oral treatments for RRMS, dimethyl fumarate (DMF). Chapter 1 provides an introduction to MS and explores the need for a stratified approach to treatment. Chapter 2 outlines the materials and methods used in this study including a discussion of modelling approaches that are used for data reduction. In Chapter 3, I aimed to discriminate MS patients from healthy controls using multi-omics profiling. The RRMS patients showed greater expression of immune pathway genes, as well as raised concentrations of lipids within lipoprotein sub-fractions, relative to healthy controls. The lipid measures were predictive of disability as measured using the Expanded Disability Status Scale (EDSS) when combined in a multivariate regression model. In Chapter 4, I tested whether multi-omics profiling could further elucidate the pharmacodynamic actions of dimethyl fumarate (DMF), a disease modifying treatment for RRMS. Comparisons of patient samples pre- and 6 weeks post- initiation of DMF revealed transcriptome changes enriched for activation of nuclear factor (erythroid-derived 2)-like 2 (Nrf2) and inhibition of nuclear factor κB (NFκB). Metabolomics profiling defined elevated levels of tricarboxylic acid metabolites, fumarate, succinate, succinyl-carnitine and methyl-succinylcarnitine. In Chapter 5, I used my prospective longitudinal data to test whether gene expression and metabolite changes associated with drug action in the blood mononuclear cell fraction at 6 weeks are associated with clinical and radiological responses at 15 months. Patients responding to treatment (measured using the composite outcome measure ‘no evidence of disease activity’) showed robust transcriptome changes between baseline and 6-weeks that were not present in non-responders. They also showed a relative stabilisation of gene expression over the remaining study period. My study thus provides evidence that multi-omics profiling could be a useful tool for stratified medicine in MS. It promises to elucidate differences that exist between disease and healthy states, further understanding of the pharmacodynamics of treatments and can provide longitudinal measures of response for monitoring the impact of a medicine. The latter could be used to optimise treatment choice for individual patients. If these methods were reduced to practice they could increase the chances of better clinical outcomes whilst avoiding otherwise unnecessary adverse events.
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Hyland, Megan H. "Impact of Race on Use of Disease-Modifying Therapy in Multiple Sclerosis". Case Western Reserve University School of Graduate Studies / OhioLINK, 2013. http://rave.ohiolink.edu/etdc/view?acc_num=case1355951207.

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Ghadiri, Mahtab. "Peripheral Immune Phenotyping in Multiple Sclerosis: Immunomodulatory Treatment Effects and Treatment Response Biomarkers". Thesis, The University of Sydney, 2019. http://hdl.handle.net/2123/20809.

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Disease modifying therapies (DMTs) used in the treatment of relapsing remitting multiple sclerosis (RRMS) have broad effects on the immune system that are incompletely understood. There is great heterogeneity in treatment response to most DMTs. However, biomarkers predicting treatment response are lacking. In this thesis, the peripheral immune changes induced by treatment with two DMTs, fingolimod (FTY) and dimethyl fumarate (DMF), are examined in detail by immune phenotyping using multicolour flow cytometry. Chapter 3 presents a longitudinal study of T cell subsets in patients commencing treatment with DMF. Differential losses of T cell subsets are found, including relative changes in regulatory and effector subsets potentially relevant to the mechanism of action of DMF. DMF-induced lymphopaenia is further studied in an in vitro culture system. The study results suggest that differential susceptibility of distinct T cell subsets to DMF-induced apoptosis may underly differential T cell losses seen in treated patients. In Chapter 4, the effects of FTY on peripheral T cell subsets and the reversibility of these effects is explored in patients ceasing FTY treatment. Long-lasting alterations in circulating T cell subsets are documented, indicating that FTY-induced changes in the peripheral immune repertoire may persist beyond the time taken for clinical laboratory measures to normalise. Chapter 5 presents a longitudinal study of a broad range of immune cell subsets in patients commencing FTY. Changes in potentially disease-relevant immune cell subsets not previously examined in FTY-treated patients are documented. Using magnetic resonance imaging (MRI) and clinical information to assess disease activity in these patients, potential immune biomarkers of FTY treatment response are uncovered. This thesis expands on our understanding of the effects of MS DMTs on the peripheral immune repertoire and highlights the utility of immune phenotyping in exploring DMT mechanisms of action and treatment response biomarkers.
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Zimmer, Andrea, Michael Coslovsky, Ivo Abraham y Bernhard F. Décard. "Adherence to fingolimod in multiple sclerosis: an investigator-initiated, prospective, observational, single-center cohort study". DOVE MEDICAL PRESS LTD, 2017. http://hdl.handle.net/10150/626094.

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Objectives: Adherence to multiple sclerosis (MS) treatment is essential to optimize the likelihood of full treatment effect. This prospective, observational, single-center cohort study investigated adherence to fingolimod over the 2 years following treatment initiation. Two facets of adherence - implementation and persistence - were examined and compared between new and experienced users of disease-modifying treatments (DMTs). Materials and methods: Implementation rates were based on the proportion of days covered and calculated as percentages per half-yearly visits and over 2 years, captured through refill data, pill count, and self-report. Nonadherence was defined as taking less than 85.8% of prescribed pills. Implementation rates were classified as nonadherent (< 85.8%), suboptimally adherent (>= 85.8% but. 96.2%), and optimally adherent (>= 96.2%), including perfectly adherent (100%). Persistence, ie, time until discontinuation, was analyzed by Kaplan-Meier analysis. Reasons for discontinuation were recorded. Results: The cohort included 98 patients with relapsing MS, all of whom received a dedicated education session about their medication. Of these 80% were women, 31.6% had fingolimod as first DMT, and 68.4% had switched from other DMTs. The mean implementation rate over 2 years was 98.6% (IQR(1-3) 98.51%-98.7%) and did not change significantly over time; 89% of measurements were in the optimally adherent category, 45.6% in the perfectly adherent category. There was one single occurrence of nonadherence. New users of DMTs were 1.29 times more likely to be adherent than experienced users (OR 1.29, 95% CI 1.11-1.51; P < 0.001), but not more persistent. Nineteen of 98 patients discontinued fingolimod. Conclusion: The very high implementation rates displayed in this sample of MS patients suggest that facilitation by health care professionals in preserving adherence behavior may be sufficient for the majority of patients. Targeted interventions should focus on patients who are nonadherent or who stop treatment without intention to reinitiate.
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Betts, Matt. "Therapeutic potential of targeting group III metabotropic glutamate receptors as a disease modifying strategy in the treatment of Parkinson's disease". Thesis, King's College London (University of London), 2012. https://kclpure.kcl.ac.uk/portal/en/theses/therapeutic-potential-of-targeting-group-iii-metabotropic-glutamate-receptors-as-a-disease-modifying-strategy-in-the-treatment-of-parkinsons-disease(4771cee9-facd-4257-9e4f-1419dd4416b7).html.

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Parkinson’s disease (PD) is characterised by a progressive loss of dopaminergic neurones from the SNpc, leading to numerous downstream changes in the basal ganglia circuitry. Overactivity of the glutamatergic subthalamonigral pathway may underlie this continual degeneration of the nigrostriatal system. With this in mind, this thesis examined whether selective activation of group III metabotropic glutamate receptor subtypes may offer a novel strategy to halt persistent degeneration in PD. Initial distribution studies revealed mGlu4 and 7 group III mGlu receptor subtypes, demonstrated particularly intense immunoreactivity in the SNpc, suggesting these receptors may be ideally positioned to provide neuroprotective effects. Therefore, the first objective was to confirm this neuroprotective possibility using a broad spectrum agonist, L-AP4. Sub-chronic supranigral L-AP4 treatment mediated functional neuroprotection against a unilateral 6-OHDA lesion of the SN, confirmed by behavioural assessment and post-mortem analyses. Secondly, the pharmacological identity of the group III mGlu receptor mediating this protective effect was examined. To investigate mGlu4 receptors, the novel mGlu4 selective PAM VU0155041, was also shown to provide functional neuroprotection in the 6-OHDA rat model to an almost comparable level reached with L-AP4. Whilst these neuroprotective effects are likely mediated by an inhibition of glutamate to protect from glutamate-mediated excitotoxicity, VU015504 also led to a significant reduction in levels of GFAP and IBA-1 suggesting an additional anti-inflammatory action. Further studies revealed little evidence for co-localisation of mGlu4 receptors with GFAP in the SN suggesting this anti-inflammatory component likely reflects an indirect effect via stimulation of neuronal mGlu4 receptors. Finally, to investigate mGlu7 receptors, the selective allosteric agonist AMN082, was also shown to protect the nigrostriatal tract and demonstrate a degree of preservation of motor function. In contrast, mGlu8 receptor activation using the selective agonist DCPG, failed to protect the nigrostriatal tract or preserve motor behaviour. Collectively, these findings demonstrate that, of the group III mGlu receptors investigated, mGlu4 offers the most potential as a promising target for establishing disease modification in PD.
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Silwer, Louise. "Public Health Aspects of Pharmaceutical Prescription Patterns : Exemplified by treatments for prevention of cardiovascular disease". Doctoral thesis, Nordic School of Public Health NHV, 2007. http://urn.kb.se/resolve?urn=urn:nbn:se:norden:org:diva-3425.

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Public health aspects of pharmaceutical prescription patterns: Exemplified by treatments for prevention of cardiovascular disease. Louise Silwer. ISBN: 978-91-85721-18-4 ISSN: 0283-1961Main aim:To study patterns and trends of dispensed prescriptions, to explore what proportion of the population is exposed to some of the more prevalently prescribed pharmaceuticals, and to find possible ways of measuring drug-induced adverse symptoms in the population. Further, to illuminate conditions surrounding prescribing in primary prevention of cardiovascular disease. Methods: In three descriptive studies of prescription patterns, prescription data at aggregate level from a Swedish county were analysed retrospectively, and proportions were calculated. Data from the first ten years of the studies were obtained from a local prescription study, and data from another five years were local data from a national prescription survey. Data from a Danish database (OPED), with data at the individual level, were used for a prescription sequence symmetry analysis, and when Swedish national prescription data at the individual level became accessible, they were used for calculations of drug prevalence in the entire Swedish population. In a qualitative analysis of interview data, a phenomenographic approach was used. Main results: The purchase of pharmaceuticals on prescription almost doubled in the studied county in the period 1988-2002. Some common pharmaceuticals that increased to a great extent among the older part of the population were cardiovascular preventive drugs, such as antihypertensive and lipid modifying agents, and also hormone replacement therapy for women. In 2005, over half of all Swedish citizens, aged 60 or over, purchased antihypertensive or lipid modifying preparations during a six-month period. The different views that were found among GPs, regarding beliefs and practical management of primary prevention of CVD, could be interpreted as a reflection of the complexity of patient counselling in primary prevention in practice. Conclusion: The increase in dispensed prescriptions over the 15 years and the magnitude of the prevalence of the studied pharmaceuticals, such as antihypertensive, lipid modifying and hormonal treatments, which to a great extent are used by ‘healthy’ people, point to the need of following-up both beneficial and harmful consequences on public health. The prevalence of preventive treatments together with the variation in views of administration of primary prevention of cardiovascular disease, also point to the need of clarification of guidelines regarding pharmaceutical primary prevention and encouragement of therapy discussions among GPs.
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Mok, Simon Wing-Fai [Verfasser]. "Modifying prion disease development by ablation of galectin-3 and by simvastatin-treatment / Simon Wing-Fai Mok". Berlin : Freie Universität Berlin, 2012. http://d-nb.info/1026788455/34.

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Yue, Xiaomeng. "Medication Patterns and Comparative Effectiveness Research of Biologic Disease-modifying Antirheumatic Drugs in Children Newly Diagnosed with Juvenile Idiopathic Arthritis using Electronic Medical Records". University of Cincinnati / OhioLINK, 2020. http://rave.ohiolink.edu/etdc/view?acc_num=ucin1613751938659097.

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Chung, Gawun Jah-Hung. "Towards defining a role for zoledronate as a disease-modifying treatment in osteoarthritis : an in vitro study of the effects of zoledronate on cartilage and chondrocyte proteoglycan metabolism". Thesis, University College London (University of London), 2008. http://discovery.ucl.ac.uk/1446441/.

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Osteoarthritis (OA) is a common group of disabling joint disorders for which there are limited pharmacological therapies to alter disease progression. Zoledronate, one of several bisphosphonates found to modulate joint changes in animal OA models, may have a disease-modifying role, and potential mechanisms of action include effects on cartilage and/or subchondral bone. In OA cartilage, loss of aggrecan, the main glycosaminoglycan-bearing proteoglycan, and degradation of type II collagen are major biochemical changes arising from imbalances in matrix synthesis and degradation. Zoledronate, in common with other bisphosphonates, is capable of inhibiting matrix metallo-proteinases, enzymes implicated in OA cartilage matrix catabolism, providing a biochemical basis for cartilage effects but it is not known whether direct effects occur at the cell/tissue level. Studies described in this thesis have explored the hypothesis that zoledronate modifies cartilage metabolism to reduce cartilage glycosaminoglycan loss in OA. Short-term treatment effects on proteoglycan synthesis and degradation were examined in vitro in models of cartilage and chondrocyte metabolism, with IL-1a used to stimulate "OA-like" tissue glycosaminoglycan release. Zoledronate 10M adversely affected cell viability, proliferation and proteoglycan synthesis in bovine articular chondrocytes and, thus, was the upper limit of the concentration range investigated. No enhancing effects were observed with zoledronate 10"10M to 10/1 on proteoglycan synthesis in bovine articular chondrocytes. No effects on glycosaminoglycan release were seen with zoledronate 10"10M to 10"5M in bovine articular cartilage or with zoledronate 10"8M to "10M in alginate bead constructs containing bovine articular chondrocytes and matrix. Thus, a direct effect on cartilage proteoglycan metabolism following short-term treatment does not appear to be a mechanism of action for zoledronate as a disease-modifying treatment in OA. However, preventative or delayed treatment effects remain unaddressed and other potential targets for zoledronate in the OA joint include cartilage type II collagen metabolism and subchondral bone metabolism.
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Calocer, Floriane. "Déterminants socio-économiques et sclérose en plaques : Influence sur l'évolution et la prise en charge thérapeutique La sclérose en plaques : historique, épidémiologie et pathogénie Socioeconomic deprivation increases the risk of disability in multiple sclerosis patients Socio-economic status influences access to second-line disease modifying treatment in Relapsing Remitting Multiple Sclerosis patients". Thesis, Normandie, 2020. http://www.theses.fr/2020NORMC402.

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L’objectif de la thèse était d’étudier l’influence des déterminants socio-économiques dans l’évolution et la prise en charge thérapeutique de la Sclérose en Plaques (SEP). Dans une 1ère partie, nous avons réalisé une revue de la littérature afin d’identifier l’association entre les déterminants socio-économiques et risque de développer une SEP. Les études les plus récentes, de bon niveau méthodologique, sur de grandes cohortes de patients mettent en avant qu’un faible niveau socio-économique serait associé à un risque plus élevé de développer une SEP. Dans une 2ème partie, nous avons étudié l’influence des déterminants socio-économiques sur le risque d’atteindre des niveaux de handicap modérés (EDSS 4) et sévères (EDSS 6) chez des patients atteints de SEP récurrente-rémittente (SEP-RR) et secondairement progressive (SEP-SP), inclus dans les bases de données de 3 centres expert SEP de l’Observatoire Français de la SEP (OFSEP), Caen, Rouen et Lille (N=3641). Comme indicateur du niveau socio-économique, nous avons utilisé l’EDI, un indice agrégé et écologique de défavorisation socio-économique. Le risque d’atteindre un EDSS 4 ou un EDSS 6 était significativement plus important chez les patients les plus défavorisés socio-économiquement (EDI-quintile 5) que chez les patients les plus favorisés (EDI-quintile 1) ; Hazard Ratio ajusté HRa=1.37 [1.15-1.64] pour le risque d’atteindre un EDSS 4 et HRa=1.42 [1.13-1.75] celui d’atteindre un EDSS 6.Enfin, dans une 3ème partie, nous avons étudié l’influence des déterminants socio-économiques approchés par l’EDI sur la prise en charge thérapeutique et plus spécifiquement l’accès aux traitements de fond (DMT), en prenant en considération l’accès au 1er DMT des patients SEP-RR et -SP inclus dans les bases de données des 3 mêmes centres de l'OFCE (N=3293) puis l’accès aux DMT de 2nde ligne (Cyclophosphamide, Mitoxantrone, Natalizumab, et Fingolimod) des patients SEP-RR de la base de donnée régionale de la Normandie Occidentale (N=733). D’après nos résultats il semble que les déterminants socio-économiques n’influencent pas l’accès aux 1er DMT. Cependant nous avons montré une influence des déterminants socio-économiques sur l’accès aux DMT de 2nde ligne. En effet, partir de 5 ans d’exposition à un 1er DMT, la probabilité de recevoir un DM de 2nde ligne est 3 fois plus élevée pour les patients socio-économiquement les plus favorisés (EDI-quintile 1) comparé aux patients avec des EDI plus élevés (EDI-quintiles 2 à 5) HRa=3.14 [1.72-5.72]. Nous avons mis en évidence que les déterminants socio-économiques influençant plusieurs moments clefs de l’évolution de la maladie et de la prise en charge thérapeutiques des patients SEP. Ces observations devraient encourager les neurologues et les équipes paramédicales à prendre des dispositions afin d’améliorer la prise en charge des patients socio-économiquement vulnérables en cherchant à réduire l’impact de ces inégalités sociales de santé (ISS) sur l’évolution de la SEP et l’accès aux soins
The general objective of the thesis was to study the influence of socio-economic determinants in Multiple Sclerosis (MS) evolution and therapeutic health care. In a 1st part, we performed a review of the literature, to identify the association between socio-economic déterminants and MS risk. The most recent studies performed with solid methodology on large cohort show that low socio-economic status seems to be associated with a higher risk of Multiple Sclerosis. In a 2nde part, we assessed the influence of socioeconomic deprivation on the risk to reach a moderate disability level (EDSS 4) and a severe disability level (EDSS 6) in RR-MS and SP-MM patients included in the databases of 3 MS expert centres (Caen, Rouen, Lille) of the French Observatory for MS (OFSEP) (N=3641). The EDI (European Deprivation Index), an ecological and aggregated indicator was used as an indicator of socio-economic level. The risk of reaching EDSS 4 and EDSS 6 for more socioeconomically deprived patients (EDI Q5) was independently higher than in the less socioeconomically deprived patients (EDI Q1), adjusted Hazard Ratio HRa=1.37 [1.15-1.64] to reach EDSS 4 and HRa=1.42 [1.13-1.75] to reach EDSS 6. Finally, in a 3rd part, we studied the influence of socio-economic determinants approached by the EDI on therapeutic health care, and specifically on the access to disease modifying treatments (DMT), taking into consideration the access to first DMT in RR-MS and SP-MS patients included in databases of the 3 same OFSEP centres (N=3293), then on the access to 2nde line DMT (Cyclophosphamide, Mitoxantrone, Natalizumab, and Fingolimod) in RR-MS patients, included in the regional database of Western-Normandy (N=733). According our results, socio-economic determinants do not seem to influence the access to a 1st DMT. However, we showed an influence of socio-economic determinants on the access to 2nde line DMT. Indeed, after 5 years from initiation of a first DMT the risk of accessing a 2nd line DMT is 3 times higher for patients with lower deprivation indices (1st quintile of EDI) HRa= 3.14 95%CI [1.72-5.72] compared to patients with higher values (EDI quintiles 2 to 5). We highlighted that socio-economic determinants influenced many key moments in evolution and therapeutic health care of MS patients. These observations encourage neurologist and paramedical team to take every provision to improve health care of socio-economically vulnerable MS patients and keep working to reduce social inequalities of health on evolution and access to care in MS
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Libros sobre el tema "Disease-modifying treatments"

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Frost & Sullivan., ed. U.S. autoimmune disease therapeutic product markets: Disease-modifying companies heading toward regulatory approval. Mountain View, CA: Frost & Sullivan, 1994.

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Nie, Pei Huey y David L. Sultzer. Treatments for Neurocognitive Disorders. Oxford University Press, 2015. http://dx.doi.org/10.1093/med:psych/9780199342211.003.0026.

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Dementia, or neurocognitive disorders, refers to a number of clinical syndromes originating in brain pathology and characterized by cognitive deficits and functional impairment. This chapter provides an update on treatment options in addition to a brief summary of dementia types and an overview of the diagnostic criteria for cognitive disorders. The diagnosis of dementia is ultimately a clinical one and includes a multidimensional perspective; as such, treatment requires a comprehensive approach. This chapter addresses two aspects of the treatment of neurocognitive disorders: pharmacological interventions that can temporarily slow the decline of cognitive deficits and the management of behavioral and psychological symptoms (neuropsychiatric symptoms) associated with dementia syndromes. The chapter also reviews disease-modifying treatments in development that may beneficially alter the course of disease, or reduce or prevent symptom expression in those at risk.
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Tsai, Po-Heng. Cognitive Enhancers for Alzheimer’s Disease. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780190214401.003.0003.

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Alzheimer’s disease (AD) is the most common cause of dementia. In the United States, an estimated 5.3 million people had AD dementia in 2015, including 200,000 individuals younger than age 65 years. The number of people who are affected by AD is projected to reach 16 million in 2050. There is a tremendous cost associated with caring for people with AD. In 2015, the direct costs to US society of caring for those with AD totaled an estimated $226 billion, and if no effective disease-modifying treatments become available, this could increase to $1.1 trillion in 2050. In addition to medical costs, in 2014, caregivers of people with AD and other dementias provided an estimated 17.9 billion hours of unpaid assistance, which translates to a value of $217.7 billion. Therefore, cognitive enhancers for AD by improving cognition could address symptoms associated with AD, reduce caregiver burden, and limit health care costs.
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Paganoni, Sabrina y Nazem Atassi. Upper Motor Neuron Disorders Hereditary Spastic Paraplegia and Primary Lateral Sclerosis. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780199937837.003.0032.

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Upper motor neuron (UMN) syndromes are a group of rare, degenerative neurological disorders that are classified as either hereditary spastic paraplegia (HSP) or primary lateral sclerosis (PLS). Our understanding of their underlying pathophysiology is unfortunately very limited and has been a significant barrier to the development of disease-modifying treatments. Recent advances in genetics and in vitro and in vivo disease modeling have provided new insights into disease mechanisms and hold the promise to lead to the future development of mechanism-based therapies.
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Rho, Jong M. Overview. Editado por Jong M. Rho. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780190497996.003.0011.

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After nearly a century of clinical use, the ketogenic diet is firmly established as an efficacious treatment for medically intractable epilepsy. Intriguingly, there is growing experimental evidence that the ketogenic diet and its metabolites also render neuroprotective and potentially disease-modifying effects. Hence, dietary and metabolic therapies have been attempted in a variety of neurological disorders other than epilepsy, including brain cancer, cognitive disorders, autism, neurotrauma, pain, and multiple sclerosis. This section, “Ketogenic Diet: Emerging Clinical Applications and Future Potential,” explores the current preclinical and clinical evidence for metabolism-based treatments designed to counter the myriad disease processes seen in many neurological conditions. Specific attention has been given to the effects of the ketogenic diet in malignant brain cancer, autism spectrum disorder, Alzheimer’s disease, traumatic brain and spinal cord injury, pain, and multiple sclerosis.
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Hopkins, Philip M. Musculoskeletal disorders and anaesthesia. Editado por Philip M. Hopkins. Oxford University Press, 2017. http://dx.doi.org/10.1093/med/9780199642045.003.0080.

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This chapter covers the anaesthetic implications of the polyarthropathies, connective tissue diseases, and primary myopathies. There are generic considerations for management of patients with joint and muscle disease but many of these conditions have multisystem involvement, the nature of which varies between the individual members of each class. Anaesthetic management of the polyarthropathies requires knowledge of the adverse effects of disease-modifying drugs and new biological treatments. Advances in genetic diagnosis of inherited myopathies aid the identification of potential hazards of anaesthesia in these challenging patients.
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Chan, Jonathan y Nigil Haroon. Treatment: NSAIDs. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780198734444.003.0020.

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Non-steroidal anti-inflammatory drugs (NSAIDs) constitute a diverse group of medications that inhibit prostaglandin synthesis. NSAIDs form the first-line pharmacological therapy in ankylosing spondylitis (AS). A number of randomized controlled trials (RCTs) support the efficacy of NSAIDs in reducing pain and improving patient function. Head-to-head comparisons have demonstrated equivalent effect of different NSAIDs in symptom control. The proposed disease-modifying potential of regular NSAID therapy is debatable and recent literature provides evidence to the contrary. Several safety concerns have been raised regarding long-term use of NSAIDs, especially an increase in cardiovascular risk. This chapter discusses the pharmacology, efficacy in treatment of AS, disease-modifying potential, and safety concerns of NSAIDs.
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Poddubnyy, Denis y Hildrun Haibel. Treatment: DMARDs. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780198734444.003.0021.

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In axial spondyloarthritis (axSpA) there is little evidence to support use of classical synthetic disease-modifying antirheumatic drugs (DMARDs), with the majority of studies performed in advanced ankylosing spondylitis. Sulfasalazine is the best investigated DMARD in axSpA. Its positive clinical effect, if any, seems to be more prominent in the presence of peripheral arthritis, although a certain proportion of patients with axial disease might benefit from sulfasalazine therapy. Available data indicate that there is no evidence that methotrexate might be effective in axial disease, and only marginal evidence exists in support of methotrexate use in case of peripheral involvement. No true disease-modifying properties (e.g. retardation of structural damage progression in the spine) have been demonstrated for DMARDs in axSpA to date. Efficacy of a combination therapy (e.g. methotrexate plus sulfasalazine) as well as benefits of methotrexate (or other DMARDs) in addition to tumour necrosis factor α‎ inhibitors in axSpA remain uncertain.
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Morgan, David. Immunotherapy for Neurodegenerative Disorders. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780190233563.003.0017.

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Neurodegenerative diseases are a growing health concern through the world as gains in longevity result in an increased population at risk of these age-related disorders. Unfortunately no disease modifying treatments exist for these disorders. Over the last three decades enormous insights have been gained into the causes of these disorders. One approach to treating these diseases is to direct immunotherapy against the misfolded proteins that accumulate within the brains of those with neurodegenerative disease in an attempt to clear the accumulating proteins and slow or prevent expression of the disease. This chapter summarizes the recent (and frustrating) experience with anti-Aβ‎ immunotherapy to treat mild to moderate Alzheimer’s disease, and holds hope that newer generation antibodies and treating presymptomatic disease will have greater impact. In addition, it reviews the preclinical data regarding approaches to treating tau, synuclein, and prion disorders, all of which demonstrate consistent effects in mice and cultured neurons.
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Cummings, Jeffrey L. y Jagan A. Pillai. Neurodegenerative Diseases. Oxford University Press, 2016. http://dx.doi.org/10.1093/med/9780190233563.003.0001.

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Neurodegenerative diseases (NDDs) are growing in frequency and represent a major threat to public health. Advances in scientific progress have made it clear that NDDs share many underlying processes, including shared intracellular mechanisms such as protein misfolding and aggregation, cell-to-cell prion-like spread, growth factor signaling abnormalities, RNA and DNA disturbances, glial cell changes, and neuronal loss. Transmitter deficits are shared across many types of disorders. Means of studying NDDs with human iPS cells and transgenic models are similar. The progression of NDDs through asymptomatic, prodromal, and manifest stages is shared across disorders. Clinical features of NDDs, including cognitive impairment, disease progression, age-related effects, terminal stages, neuropsychiatric manifestations, and functional disorders and disability, have many common elements. Clinical trials, biomarkers, brain imaging, and regulatory aspects of NDD can share information across NDDs. Disease-modifying and transmitter-based therapeutic interventions, clinical trials, and regulatory approaches to treatments for NDDs are also similar.
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Capítulos de libros sobre el tema "Disease-modifying treatments"

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Bespalov, Anton, Corinna Klein, Berthold Behl, Gerhard Gross y Hans Schoemaker. "Development of Disease-Modifying Treatment of Schizophrenia". En Novel Antischizophrenia Treatments, 419–42. Berlin, Heidelberg: Springer Berlin Heidelberg, 2012. http://dx.doi.org/10.1007/978-3-642-25758-2_14.

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Hutchinson, Michael. "Current Treatments for Progressive Multiple Sclerosis: Disease-Modifying Therapies". En Progressive Multiple Sclerosis, 187–219. London: Springer London, 2012. http://dx.doi.org/10.1007/978-1-4471-2395-8_9.

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Jayne, David. "Intravenous immunoglobulin as immuno-modifying treatment". En Disease-modifying Therapy in Vasculitides, 81–111. Basel: Birkhäuser Basel, 2001. http://dx.doi.org/10.1007/978-3-0348-8235-4_5.

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Schmitt, Wilhelm H., Ernst C. Hagen y Fokko J. van der Woude. "T-cell directed treatment: anti-thymocyte globulin". En Disease-modifying Therapy in Vasculitides, 113–23. Basel: Birkhäuser Basel, 2001. http://dx.doi.org/10.1007/978-3-0348-8235-4_6.

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Stone, John H. "Tumor necrosis factor (TNF) inhibition in the treatment of vasculitis". En Disease-modifying Therapy in Vasculitides, 41–63. Basel: Birkhäuser Basel, 2001. http://dx.doi.org/10.1007/978-3-0348-8235-4_3.

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Guillevin, Loïc. "Interferon-α for the treatment of virus-related systemic vasculitides". En Disease-modifying Therapy in Vasculitides, 147–57. Basel: Birkhäuser Basel, 2001. http://dx.doi.org/10.1007/978-3-0348-8235-4_8.

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Boomsma, Maarten M., Coen A. Stegeman, Cees G. M. Kallenberg y Jan W. Cohen Tervaert. "Prevention of relapsing disease in anti-neutrophil cytoplasmic antibody related necrotizing small-vessel vasculitis: the role for autoantibody guided and anti-bacterial treatment". En Disease-modifying Therapy in Vasculitides, 181–99. Basel: Birkhäuser Basel, 2001. http://dx.doi.org/10.1007/978-3-0348-8235-4_10.

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Rizvi, Syed A. "Disease Modifying Agents in the Treatment of Multiple Sclerosis". En Clinical Neuroimmunology, 131–56. Totowa, NJ: Humana Press, 2011. http://dx.doi.org/10.1007/978-1-60327-860-7_7.

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Tzioufas, Athanasios G. y Haralampos M. Moutsopoulos. "Current Treatment of Extraglandular Manifestations with Disease-Modifying and Immunosuppressive Agents". En Sjögren’s Syndrome, 337–44. New York, NY: Springer New York, 2011. http://dx.doi.org/10.1007/978-1-60327-957-4_19.

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Herrero-Hernandez, Pablo, Atze J. Bergsma y W. W. M. Pim Pijnappel. "Generation of Human iPSC-Derived Myotubes to Investigate RNA-Based Therapies In Vitro". En Methods in Molecular Biology, 235–43. New York, NY: Springer US, 2022. http://dx.doi.org/10.1007/978-1-0716-2010-6_15.

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AbstractAlternative pre-mRNAsplicing can be cell-type specific and results in the generation of different protein isoforms from a single gene. Deregulation of canonical pre-mRNAsplicing by disease-associated variants can result in genetic disorders. Antisense oligonucleotides (AONs) offer an attractive solution to modulate endogenous gene expression through alteration of pre-mRNAsplicing events. Relevant in vitro models are crucial for appropriate evaluation of splicing modifying drugs. In this chapter, we describe how to investigate the splicing modulating activity of AONs in an in vitro skeletal muscle model, applied to Pompe disease. We also provide a detailed description of methods to visualize and analyze gene expression in differentiated skeletal muscle cells for the analysis of muscledifferentiation and splicing outcome. The methodology described here is relevant to develop treatment options using AONs for other genetic muscle diseases as well, including Duchenne muscular dystrophy, myotonic dystrophy, and facioscapulohumeral muscular dystrophy.
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Actas de conferencias sobre el tema "Disease-modifying treatments"

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Butler, D., I. Makariou, M. Scoto, V. Gowda, M. Fernandez, E. Wraige, H. Jungbluth, F. Muntoni, H. Tan y F. Trucco. "Sleep outcomes in spinal muscular atrophy and role of disease-modifying treatments." En ERS International Congress 2022 abstracts. European Respiratory Society, 2022. http://dx.doi.org/10.1183/13993003.congress-2022.1516.

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Salvador Llana, I., S. Álvarez Atienza, AM Martín De Rosales Cabrera, L. Borrega Canelo y M. Pérez Encinas. "4CPS-333 Treating multiple sclerosis patients with infusion of disease modifying treatments during the COVID-19 pandemic". En 25th Anniversary EAHP Congress, Hospital Pharmacy 5.0 – the future of patient care, 23–28 March 2021. British Medical Journal Publishing Group, 2021. http://dx.doi.org/10.1136/ejhpharm-2021-eahpconf.165.

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Oprea, SM y S. Negres. "4CPS-131 Switching and discontinuation of disease-modifying treatments in multiple sclerosis patients: experience in a university hospital". En 26th EAHP Congress, Hospital pharmacists – changing roles in a changing world, 23–25 March 2022. British Medical Journal Publishing Group, 2022. http://dx.doi.org/10.1136/ejhpharm-2022-eahp.153.

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Requião, Letícia Escorse, Giulia Freitas, Mayanna Macedo, Hanny Gondim, Blenda Antunes y Bruno Lopes. "Monoclonal antibodies: a new trend for the treatment of Alzheimer’s disease?" En XIII Congresso Paulista de Neurologia. Zeppelini Editorial e Comunicação, 2021. http://dx.doi.org/10.5327/1516-3180.439.

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Introduction: Alzheimer’s disease (AD) is the main form of senile dementia. Most of the supposedly disease-modifying treatments in development are directed against the β-amyloid peptide, the administration of exogenous anti-Aβ monoclonal antibodies is a passive immunization strategy aimed at resolving the aggregation of this substance. Objective: Analyze the effectiveness of monoclonal antibodies in the treatment of Alzheimer’s disease. Methods: This is a literature review, based on randomized clinical trials published between 2014 and 2021. The search was conducted in the PubMed database. Results: According to the eligibility criteria, 10 articles were selected. Two of the randomized, double-blind, placebo-controlled phase III studies, one published in 2018 and the other published in 2016, evaluated the intervention with Solanezumab and Bapineuzumab, respectively. Both were not shown to be statistically significant (P = 0.10) for the outcome improvement of the score in the cognitive subscale of 14 and 11 items “Alzheimer’s Disease Assessment Scale” (ADAS-cog14 / 11). However, in a phase II randomized placebo-controlled clinical trial, published in 2021, the use of Donanemab in patients with early Alzheimer’s disease resulted in statistically significant cognitive and functional improvement (P = 0.04) for the outcome change in the scale “Integrated Alzheimer’s Disease Rating” (iADR). Conclusion: Although the use of Donanemab has resulted in cognitive and functional improvement, randomized, double-blind, placebo-controlled, phase III clinical trials need to be conducted to prove the efficacy and safety of its use in clinical practice. Other monoclonal antibodies evaluated did not demonstrate evidence of benefit.
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Weinblatt, M. "SP0060 Disease modifying drugs in the treatment of rheumatoid arthritis". En Annual European Congress of Rheumatology, Annals of the rheumatic diseases ARD July 2001. BMJ Publishing Group Ltd and European League Against Rheumatism, 2001. http://dx.doi.org/10.1136/annrheumdis-2001.17.

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Rodrigues, M., A. Soares, P. Almeida, A. Armando, M. Vargas Gomes, V. Andreozzi y J. Félix. "6ER-009 Treatment patterns in multiple sclerosis with disease modifying drugs". En 25th EAHP Congress, 25th–27th March 2020, Gothenburg, Sweden. British Medical Journal Publishing Group, 2020. http://dx.doi.org/10.1136/ejhpharm-2020-eahpconf.444.

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Monet, M., C. Prati, X. Guillot, M. Sondag, F. Verhoeven, F. Aubin y D. Wendling. "SAT0606 Disease modifying anti rheumatic drugs in the treatment of sapho syndrome: systematic literature analysis". En Annual European Congress of Rheumatology, EULAR 2018, Amsterdam, 13–16 June 2018. BMJ Publishing Group Ltd and European League Against Rheumatism, 2018. http://dx.doi.org/10.1136/annrheumdis-2018-eular.2714.

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Nasef, S. I., G. El Deriny, M. Eissa, M. A. Mortada, W. Hassan, D. Mekkawy y Y. El Miedany. "THU0603 Disease modifying anti-rheumatic drugs in juvenile idiopathic arthritis: moving towards targeted individualised treatment strategy". En Annual European Congress of Rheumatology, EULAR 2018, Amsterdam, 13–16 June 2018. BMJ Publishing Group Ltd and European League Against Rheumatism, 2018. http://dx.doi.org/10.1136/annrheumdis-2018-eular.4156.

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Feldman, C. H., J. Lii, C. Gopalakrishnan, J. M. Franklin y S. C. Kim. "THU0664 Treatment adherence to conventional and biologic disease modifying antirheumatic drugs in patients with rheumatoid arthritis". En Annual European Congress of Rheumatology, EULAR 2018, Amsterdam, 13–16 June 2018. BMJ Publishing Group Ltd and European League Against Rheumatism, 2018. http://dx.doi.org/10.1136/annrheumdis-2018-eular.5544.

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Arancón Pardo, A., C. Sobrino Jiménez, E. Rodríguez Martín, C. Bilbao Gómez-Martino, E. Villamañán Bueno, I. Jiménez-Nácher, F. Moreno Ramos et al. "4CPS-109 Analysis of changes in disease modifying treatment in the management of patients with multiple sclerosis". En 25th EAHP Congress, 25th–27th March 2020, Gothenburg, Sweden. British Medical Journal Publishing Group, 2020. http://dx.doi.org/10.1136/ejhpharm-2020-eahpconf.210.

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