Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Recruiting
N/A
Interventional Study
Spinal Muscular Atrophy
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
At a Glance
- Age
- 0 – 16
- Sex
- Any
- Study type
- Interventional
- Purpose
- Other
- Participants needed
- 60 (estimated)
- Sponsor
- Hospices Civils de Lyon · Other
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About This Trial
With the advent of new treatments for ASI, new phenotypes are emerging. The investigators propose to describe these new phenotypes by prospectively following children with ASI of all types treated with TRS and aged under 16 for 2 years.
The investigators also propose to evaluate potential assessment tools to determine whether they are relevant for monitoring this population, either routinely or f…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Genetically confirmed infantile or juvenile spinal muscular atrophy
* Treated with a therapy that restores SMN protein expression (e.g. nusinersen, risdiplam, onasemnogene abeparvovec)
* Aged 0 to 15 years inclusive
* Informed consent signed by both parent(s)/legal guardian(s)…
Contacts