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The Natural History and Muscle Fatigability of Patients With Congenital Myopathies.
Sponsor: Radboud University Medical Center
Summary
Core myopathies (CCD/MmD), nemaline myopathies (NEM) and centronuclear myopathies (CNM) are three types of rare congenital myopathies. Not much is known about the natural history and no curative treatment is available for these groups. Also patients report fatigability as one of their symptoms. The goal of this observational study is to study the natural history during 24 months to achieve trial readiness and to study the muscle fatigability in CCD/MmD, NEM and CNM.
Official title: Trial Readiness and Trial Fitness for Congenital Myopathies: a 2-year Prospective Natural History Study Including a Cross-sectional Study on Muscle Fatigability
Key Details
Gender
All
Age Range
2 Years - Any
Study Type
OBSERVATIONAL
Enrollment
100
Start Date
2024-03-28
Completion Date
2026-11
Last Updated
2024-08-19
Healthy Volunteers
No
Interventions
Natural history and non therapeutical therapy
This study concerns a natural history study part and a muscle fatigability part. For the natural history study no interventions will be used. For the muscle fatigability part non therapeutical therapies will be used. This includes endurance tests, isokinetic dynamometry and repetitive nerve stimulation.
Locations (2)
Radboudumc
Nijmegen, Gelderland, Netherlands
UMC Utrecht
Utrecht, Netherlands