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

Jennifer M KwonKapil AryaNancy KuntzHan C PhanCory SieburgKathryn J SwobodaAravindhan VeerapandiyanBeverly AssmanSilvia Bader-WederTravis L DickendesherJennifer HansenHelen LinYing YanVamshi K Raonull null

Published in: Annals of clinical and translational neurology (2022)

In the EAP, the safety profile of risdiplam was similar to what was reported in pivotal risdiplam clinical trials. These safety data provide further support for the use of risdiplam in the treatment of adult and pediatric patients with SMA.

Keyphrases

clinical trial
quality improvement
randomized controlled trial
childhood cancer
combination therapy
young adults
replacement therapy
data analysis
phase iii