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

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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: 22 Mar 2025

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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

  • 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
  • 14 Oct 2024 According to Genentech media release, positive two-year data from the ongoing RAINBOWFISH study at the 29th World Muscle Society (WMS) Congress, 8-12 Oct, 2024, assessing the efficacy and safety of Evrysdi (risdiplam) in children with SMA who were treated pre-symptomatically as infants before six weeks of age (n=23).

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