Trial Profile

AN OPEN-LABEL STUDY OF RISDIPLAM IN INFANTS WITH GENETICALLY DIAGNOSED AND PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY

Status: Active, no longer recruiting

Phase of Trial: Phase II

Latest Information Update: 17 Mar 2026

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At a glance

Drugs Risdiplam (Primary)
Indications Spinal muscular atrophy
Focus Registrational; Therapeutic Use
Acronyms RAINBOWFISH
Sponsors Roche

Most Recent Events

26 Jan 2026 Planned End Date changed from 31 Mar 2027 to 28 Feb 2027.
17 Mar 2025 According to a Roche media release, company will present new data at the Muscular Dystrophy Association (MDA) conference, 16-19 March, 2025, in Dallas, Texas.
14 Oct 2024 Results published in a Genentech media release

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