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French Register of Patients With Spinal Muscular Atrophy
Sponsor: Assistance Publique - Hôpitaux de Paris
Summary
The primary objectives of the study are to obtain clinically meaningful data on survival and outcomes of all the patients with spinal muscular atrophy (SMA) 5q types 1 through 4 (according to international classification), being followed in the reference centers of the disease in France between September 1, 2016 and August 31, 2024. The registry will collect retrospectively and prospectively the longitudinal data of the long-term follow-up for child and adult patients, under real life conditions of current medical practice, in order to document the clinical evolution of patients (survival, motor, respiratory, orthopedic and nutritional), the conditions of use of the treatments, the mortality rates of treated and untreated patients, the tolerance of the treatments, adverse events in order to better define their places in the therapeutic strategy.
Key Details
Gender
All
Age Range
0 Days - Any
Study Type
OBSERVATIONAL
Enrollment
1000
Start Date
2020-01-31
Completion Date
2029-01-31
Last Updated
2025-04-06
Healthy Volunteers
No
Conditions
Locations (1)
Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, (French neuromuscular reference network (FILNEMUS), Hôpital Raymond Poincaré,
Garches, France