French Registry for Patients with Spinal Muscular Atrophy
French Register of Patients With Spinal Muscular Atrophy
Recruiting
Observational Study
Spinal Muscular Atrophy
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Age
- 0 Days and older
- Sex
- Any
- Study type
- Observational
- Participants needed
- 1,000 (estimated)
- Sponsor
- Assistance Publique - Hôpitaux de Paris · Other
Who this trial is looking for
This trial is looking for patients with spinal muscular atrophy (SMA) types 1 to 4 who are being treated in France. Participants will help researchers gather information about how the disease affects them and the treatments they receive over time.
Are You a Good Fit for This Trial?
You may be able to join if
- I have been diagnosed with SMA 5q types 1 to 4.
- I am being treated in a reference center in France.
- I have signed an informed consent form (for prospective study).
- I am covered by health insurance.
You may not be able to join if
- I have another type of SMA (not 5q).
- I am under guardianship or curatorship.
- I cannot understand the French language.
Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
Check Your Eligibility
About This Trial
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 l…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Genetically confirmed SMA 5q patients through 1 to 4 types treated in reference centers in France between September 1, 2016 and August 31, 2024.
* for prospective study: inform consent form signed by patient.
* for retrospective data: information form with non-opposition have …
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