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Atrophy clinical trials

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NCT ID: NCT04256265 Approved for marketing - Clinical trials for Muscular Atrophy, Spinal

An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)

Start date: n/a
Phase:
Study type: Expanded Access

This expanded access program (EAP) will provide access to risdiplam for eligible participants with Type 1 or Type 2 spinal muscular atrophy (SMA) before it is commercially available in the United States for the indication of SMA.

NCT ID: NCT03955679 Approved for marketing - Clinical trials for Spinal Muscular Atrophy

AveXis Managed Access Program Cohort for Access to AVXS-101

Start date: n/a
Phase:
Study type: Expanded Access

The purpose of this Cohort Treatment Protocol will allow access to AVXS-101 for eligible patients diagnosed with SMA.