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: 19 Mar 2024
Price :
$35
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At a glance
- Drugs Risdiplam (Primary)
- Indications Spinal muscular atrophy
- Focus Registrational; Therapeutic Use
- Acronyms RAINBOWFISH
- Sponsors Roche
- 07 Nov 2023 Planned End Date changed from 21 Jan 2029 to 31 Mar 2027.
- 04 Oct 2023 According to Roche media release, the data were presented at the 28th World Muscle Society (WMS) Congress, 3-7 October 2023.
- 04 Oct 2023 Results presented in in Roche Media Release.