Trials / Approved For Marketing
Approved For MarketingNCT04256265
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
An Expanded Access Program for Risdiplam in Patients With Type 1 or Type 2 Spinal Muscular Atrophy
- Status
- Approved For Marketing
- Phase
- —
- Study type
- Expanded Access
- Enrollment
- —
- Sponsor
- Genentech, Inc. · Industry
- Sex
- All
- Age
- 2 Months
- Healthy volunteers
- —
Summary
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.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | Risdiplam | Risdiplam will be administered orally once daily |
Timeline
- First posted
- 2020-02-05
- Last updated
- 2020-10-05
Locations
29 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT04256265. Inclusion in this directory is not an endorsement.