New Clinical Outcome Measures to Remotely Evaluate Patients With FacioScapuloHumeral Muscular Dystrophy (PROGRESS-FSHD)
Type 1 Facioscapulohumeral Muscular Dystrophy
About this trial
This is an interventional other trial for Type 1 Facioscapulohumeral Muscular Dystrophy
Eligibility Criteria
Inclusion criteria: Genetically confirmed FSHD1 or clinical diagnosis of FSHD with characteristic findings on exam and an affected parent or offspring (40) Age 18-75 years Symptomatic limb weakness Patient able to walk alone or with a walking aid. Patient affiliated to the social security system Patient giving written consent after written and oral information. If taking over the counter supplements willing to remain consistent with supplement regimen throughout the course of the study Non inclusion criteria: Patients with comorbidity not related to the disease that can modify the natural evolution of the disease or would interfere with safe testing in the opinion of the Investigator Regular use of available muscle anabolic/catabolic agents such as corticosteroids, oral testosterone or derivatives, or oral beta agonists Use of an experimental drug in an FSHD clinical trial within the past 30 days Pregnant or nursing women for women of childbearing age Patient protected by law, under guardianship or curator ship, or not able to participate in a clinical study according to the article L.1121-16 of the French Public Health Code
Sites / Locations
- CHRU de Lille
- Institut de MyologieRecruiting
- CHU de NiceRecruiting
Arms of the Study
Arm 1
Other
Patient with type 1 facioscapulohumeral muscular dystrophy