Roche Reports Results of Risdiplam in FIREFISH and SUNFISH Studies for Spinal Muscular Atrophy

 Roche Reports Results of Risdiplam in FIREFISH and SUNFISH Studies for Spinal Muscular Atrophy

Roche Reports Results of Risdiplam in FIREFISH and SUNFISH Studies for Spinal Muscular Atrophy

Shots:

  • The FIREFISH, Part 1 (dose-escalation study) & 2 studies involve assessing of Risdiplam in 21 & 41 infants with Type 1 SMA respectively. Combined results: infants sit with/without support (64.7%); median duration of treatment (14.8mos.); event-free survival overall (85.7%) & (88.2%) in the therapeutically dosed group
  • The SUNFISH, Part 1 & 2 studies involve assessing of Risdiplam vs PBO in children and young adults with Type 2 or 3 SMA aged 2–25 yrs. Combined results: @12mos. increment in SMN protein> 2-folds; improvement in MFM32 scale by 3points (58%) including 71% & 42% patients with 2-11 & 12-25yrs. respectively, no treatment-related withdrawal is observed
  • Risdiplam is an oral SMN2 splicing modifier, being evaluated for patients with SMA aged 1month to 60yrs. and is developed in collaboration with SMA Foundation and PTC Therapeutics

Click here to read full press release/ article | Ref: Roche | Image:  Behance

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