Lessons Learned From Translational Research in Neuromuscular Diseases: Impact on Study Design, Outcome Measures and Managing Expectation.
duchenne and becker muscular dystrophy
inclusion criteria
matching criteria
spinal muscular atrophy
translational research
trial design
Journal
Frontiers in genetics
ISSN: 1664-8021
Titre abrégé: Front Genet
Pays: Switzerland
ID NLM: 101560621
Informations de publication
Date de publication:
2021
2021
Historique:
received:
17
08
2021
accepted:
09
11
2021
entrez:
23
1
2023
pubmed:
7
12
2021
medline:
7
12
2021
Statut:
epublish
Résumé
Spinal Muscular Atrophy (SMA) and Duchenne Muscular Dystrophy (DMD), two of the most common, child onset, rare neuromuscular disorders, present a case study for the translation of preclinical research into clinical work. Over the past decade, well-designed clinical trials and innovative methods have led to the approval of several novel therapies for SMA and DMD, with many more in the pipeline. This review discusses several features that must be considered during trial design for neuromuscular diseases, as well as other rare diseases, to maximise the possibility of trial success using historic examples. These features include well-defined inclusion criteria, matching criteria, alternatives to placebo-controlled trials and the selection of trial endpoints. These features will be particularly important in the coming years as the investigation into innovative therapy approaches for neuromuscular diseases continues.
Identifiants
pubmed: 36687260
doi: 10.3389/fgene.2021.759994
pii: 759994
pmc: PMC9855753
doi:
Types de publication
Journal Article
Review
Langues
eng
Pagination
759994Informations de copyright
Copyright © 2021 Stimpson, Chesshyre, Baranello and Muntoni.
Déclaration de conflit d'intérêts
GB has received consultancy honoraria from AveXis, Roche, Biogen, PTC, and Sarepta Therapeutics. GB has received speaker honoraria from AveXis, Roche and PTC. FM is supported by the NIHR Great Ormond Street Hospital Biomedical Research Centre and has received speaker and consultancy honoraria from Sarepta Therapeutics, Avexis, PTC Therapeutics, Roche and Pfizer. The remaining authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.
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