Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
|Study Type:||Expanded Access What is Expanded Access?|
|Official Title:||Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)|
- ISIS 396443
Availability of nusinersen at an existing clinical trial site will depend on territory eligibility. Program opening date will depend on regulatory requirements and center-specific factors. Participating sites will be added as they apply for the EAP. A doctor must decide whether the potential benefit outweighs the risk of receiving an investigational therapy based on the individual patient's medical history and program eligibility criteria.
The Expanded Access Program in the United States is now closed to new patient enrollment as nusinersen has received FDA approval.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02865109
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|Study Director:||Medical Director||Biogen|