Innovative research methods for studying treatments for rare diseases: methodological review.
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Systematic reviews in paediatric multiple sclerosis and Creutzfeldt-Jakob disease exemplify shortcomings in methods used to evaluate therapies in rare conditionsOffering patients more: how the West Africa Ebola outbreak can shape innovation in therapeutic research for emerging and epidemic infectionsAggregated N-of-1 trials for unlicensed medicines for small populations: an assessment of a trial with ephedrine for myasthenia gravis.Hartung-Knapp-Sidik-Jonkman approach and its modification for random-effects meta-analysis with few studiesHow do you design randomised trials for smaller populations? A framework.Combination Therapies for Traumatic Brain Injury: Retrospective ConsiderationsClinical trial design for rare cancers: why a less conventional route may be requiredDegenerative Ataxias: challenges in clinical research.How a Clinical Trial Unit can improve independent clinical research in rare tumors: the Italian Sarcoma Group experience.Protocol for a systematic review of N-of-1 trial protocol guidelines and protocol reporting guidelines.Good-quality research in rare diseases: trials and tribulations.Is my patient actually getting better? Application of the McNemar test for demonstrating the change at a single subject level.Meta-analysis of few small studies in orphan diseases.Nutritional interventions in primary mitochondrial disorders: Developing an evidence base.Treatment outcome in early diffuse cutaneous systemic sclerosis: the European Scleroderma Observational Study (ESOS).Proceedings of the COST action BM1407 inaugural conference BEAT-PCD: translational research in primary ciliary dyskinesia - bench, bedside, and population perspectives.Evaluation of pre-symptomatic nitisinone treatment on long-term outcomes in Tyrosinemia type 1 patients: a systematic reviewPhysical Therapy and Exercise Interventions in Huntington's Disease: A Mixed Methods Systematic Review.A systematic literature review of evidence-based clinical practice for rare diseases: what are the perceived and real barriers for improving the evidence and how can they be overcome?Establishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children: study protocol for systematic reviews and Delphi surveys.Expert Opinion vs Patient Perspective in Treatment of Rare Disorders: Tooth Removal in Lesch-Nyhan Disease as an Example.Handling variability and incompleteness of biological data by flexible nets: a case study for Wilson disease.An overview of the impact of rare disease characteristics on research methodology.A multicenter, retrospective medical record review of X-linked myotubular myopathy: The recensus study.Treatable inherited rare movement disorders.When and how should patients with multiple endocrine neoplasia type 1 be screened for thymic and bronchial carcinoid tumours?Long-term outcomes of daily oral vs. pulsed intravenous cyclophosphamide in a non-trial setting in ANCA-associated vasculitis.Transformative therapies for rare CFTR missense alleles.Positive Effectiveness of Tafamidis in Delaying Disease Progression in Transthyretin Familial Amyloid Polyneuropathy up to 2 Years: An Analysis from the Transthyretin Amyloidosis Outcomes Survey (THAOS).Characteristics of clinical trials in rare vs. common diseases: A register-based Latvian study.Using a meta-narrative literature review and focus groups with key stakeholders to identify perceived challenges and solutions for generating robust evidence on the effectiveness of treatments for rare diseases.Exon skipping for Duchenne muscular dystrophy: a systematic review and meta-analysis.'Quality in, quality out', a stepwise approach to EBM for rare diseases promoted by MEN1Sirolimus is efficacious in treatment for extensive and/or complex slow-flow vascular malformations: a monocentric prospective phase II studyAn American Association for the Surgery of Trauma (AAST) prospective multi-center research protocol: outcomes of urethral realignment versus suprapubic cystostomy after pelvic fracture urethral injury
P2860
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P2860
Innovative research methods for studying treatments for rare diseases: methodological review.
description
2014 nî lūn-bûn
@nan
2014年の論文
@ja
2014年学术文章
@wuu
2014年学术文章
@zh-cn
2014年学术文章
@zh-hans
2014年学术文章
@zh-my
2014年学术文章
@zh-sg
2014年學術文章
@yue
2014年學術文章
@zh
2014年學術文章
@zh-hant
name
Innovative research methods fo ...... seases: methodological review.
@en
type
label
Innovative research methods fo ...... seases: methodological review.
@en
prefLabel
Innovative research methods fo ...... seases: methodological review.
@en
P2093
P2860
P356
P1433
P1476
Innovative research methods fo ...... seases: methodological review.
@en
P2093
Joshua J Gagne
Kelly O'Keefe
Lauren Thompson
P2860
P356
10.1136/BMJ.G6802
P407
P577
2014-11-24T00:00:00Z