A Long-term Safety Study in Brazilian Patients With a Diagnosis of Spinal Muscular Atrophy Treated With Zolgensma

Recruiting Observational Study
Spinal Muscular Atrophies
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.
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At a Glance
Age
0 – 100
Sex
Any
Study type
Observational
Participants needed
50 (estimated)
Sponsor
Novartis Pharmaceuticals · Industry
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About This Trial

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Subject's parent or legal guardian has provided signed eICF. * Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene. * Subject treated\* with Onasemnogene Abeparvovec (Zo…
Contacts

Novartis Pharmaceuticals

+41613241111

novartis.email@novartis.com

CONTACT

Novartis Pharmaceuticals

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