An expanded access program of risdiplam for patients with Type 1 or 2 spinal muscular atrophy.

ABSTRACT:

Objective

The US risdiplam expanded access program (EAP; NCT04256265) was opened to provide individuals with Type 1 or 2 spinal muscular atrophy (SMA) who had no satisfactory treatment options access to risdiplam prior to commercial availability. The program was designed to collect safety data during risdiplam treatment.

Methods

Patients were enrolled from 23 non-preselected sites across 17 states and treated with risdiplam orally once daily. Eligible patients had a 5q autosomal recessive Type 1 or 2 SMA diagnosis, were aged ≥2 months at enrollment, and were ineligible for available and approved SMA treatments or could not continue treatment due to a medical condition, lack/loss of efficacy, or the COVID-19 pandemic.

Results

Overall, 155 patients with Type 1 (n = 73;

SUBMITTER: Kwon JM 

PROVIDER: S-EPMC9186129 | biostudies-literature | 2022 Jun

REPOSITORIES: biostudies-literature