Condition: Spinal Muscular Atrophy · Sponsor: Assistance Publique - Hôpitaux de Paris
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.
This description comes directly from the study's public registry record.
Susana Quijano-Roy, MD, PhD · +33147107890 · susana.quijano-roy@aphp.fr
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| Unité neuromusculaire, Service de Neurologie et Réanimation Pédiatrique, (French neuromuscular reference network (FILNEMUS), Hôpital Raymond Poincaré, | Garches, France | Recruiting |
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Source record: clinicaltrials.gov/study/NCT04177134