Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)
Recruiting
Observational Study
Spinal Muscular Atrophy (SMA)
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
No Study Drug
Researchers observe your health over time — no experimental treatment is given.
At a Glance
- Sex
- Any
- Study type
- Observational
- Participants needed
- 700 (estimated)
- Sponsor
- Novartis Pharmaceuticals · Industry
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About This Trial
Spinal muscular atrophy (SMA) is a neurogenetic disorder caused by a loss or mutation in the survival motor neuron 1 gene (SMN1) on chromosome 5q13, which leads to reduced SMN protein levels and a selective dysfunction of motor neurons. SMA is an autosomal recessive, early childhood disease with an incidence of 1:10,000 live births. SMA is the leading cause of infant mortality due to genetic disea…
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Patients treated with OAV-101 with a genetically confirmed diagnosis of SMA regardless of the date of diagnosis.
* Appropriate consent/assent has been obtained for participation in the registry
Exclusion Criteria:
\- Currently enrolled in an interventional clinical trial inv…
Contacts
Novartis Gene Therapies
+4161324111
CONTACT