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

Ref: Roche | Image:  Roche