Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

Age: Not specified

Healthy Volunteers:

This study is NOT accepting healthy volunteers

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 involving an investigational medicinal product to treat SMA. Note: Patients who are participating in a Compassionate Use Program (CUP) for OAV-101 (Zolgensma) such as a Managed Access Program (MAP), an Expanded Access Program (EAP), Single Patient Investigational New Drug (IND) (SPI) or Named Patient Program (NPP) are eligible to enroll in the registry regardless of the date of a genetic or clinical diagnosis of SMA.

Interventions:

OTHER: Prospective observational registry, DRUG: Zolgensma

Conditions:

Spinal Muscular Atrophy (SMA)

Contact(s): Novartis Gene Therapies - novartis.email@novartis.com

Principal Investigator: Johnson, Nicholas, E

Phase: N/A

IRB Number: HM20015046

System ID: NCT04174157

See this study on ClinicalTrials.gov

Registry of Patients With a Diagnosis of Spinal Muscular Atrophy (SMA)

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