The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.
Roche has shared a community update to inform us about recruitment completion for their JEWELFISH clinical trial. This milestone marks the enrolment of 174 participants from across the globe. JEWELFISH is a clinical trial of the small molecule risdiplam. Risdiplam is a small molecule drug taken orally, which targets and encourages the SMN2 ‘back-up’ gene to produce a greater amount of […]
Roche’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy First placebo-controlled trial to include adults with SMA demonstrates risdiplam improved or stabilised motor function Medically meaningful and statistically significant results in primary and key secondary endpoints Pivotal SUNFISH Part 2 study population represents broad, […]
Roche announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 SMA. The primary outcome measure of the study was the proportion of infants sitting without support for at least five seconds at 12-months of treatment, assessed by the Gross Motor Scale […]
SMA Europe is pleased to share a community letter from Roche announcing a pre-approval access/ compassionate use programme for its investigational drug risdiplam. This will initially be for Type 1 SMA but will expand to Type 2 SMA upon filing for market authorisation. Dear Members of SMA Europe, Following your recent request to receive more […]
The United States’ Food and Drug Administration (FDA) has granted priority review for the New Drug Application (NDA) for risdiplam (RG7916) for the treatment of SMA. The FDA’s decision will be announced on 24th May 2020. If approved, risdiplam, an orally administered liquid, would be the first at-home administered medicine for people living with SMA. […]