A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy

Summary

A global study of oral risdiplam in pre-symptomatic participants with spinal muscular atrophy (SMA).

Timeline

Start

2019-08-07

Primary completion

2023-02-20

Completion

2027-02-28

Publications

• Finkel RS, Servais L, Vlodavets D, Zanoteli E, Mazurkiewicz-Beldzinska M, Jong YJ, Navas-Nazario A, Al-Muhaizea M, Araujo APQC, Nelson L, Wang Y, Jaber B, Gorni K, Kletzl H, Palfreeman L, Rabbia M, Summers D, Gaki E, Wagner KR, Fontoura P, Farrar MA, Bertini E; RAINBOWFISH Study Group. Risdiplam in Presymptomatic Spinal Muscular Atrophy. N Engl J Med. 2025 Aug 14;393(7):671-682. doi: 10.1056/NEJMoa2410120.

Drugs

Indications