A Prospective, Long Term Registry of Patients with a Diagnosis of Spinal Muscular Atrophy


Prospective, long term registry of patients with diagnosis of spinal muscular atrophy to assess the long term outcomes in the context of advances in treatment options.

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Not specified
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• Patients with SMA, genetically confirmed on or after 24 May 2018.
• 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 that are participating in a Compassionate Use Program (CUP) for AVXS-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 genetic confirmation of SMA.

Other: Prospective observational registry, Drug: Zolgensma

Spinal Muscular Atrophy (SMA), Rare Diseases

Natalya Alassy -
Peter Karachunski
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