SMA Newsroom

ResearchSMA EventsJun 28, 2022

11th European Conference on Rare Diseases and Orphan Products: 27th June - 1st July

The ECRD is recognised globally as the largest, patient-led rare disease policy event in which collaborative dialogue, learning and conversation takes place, forming the groundwork to shape goal-driven rare disease policies and allow for important and innovative discussions on a national and an international level to take place.

ResearchJun 27, 2022

Poster on MANATEE: Roche and Genentech clinical study of GYM329 (RO7204239) in combination with risdiplam treatment in children with SMA

MANATEE is a global, Phase 2/3 study that will assess GYM329 in combination with risdiplam in ambulant (able to walk independently) children with SMA aged 2-10 years. Children who have been previously treated with either risdiplam, nusinersen or onasemnogene abeparvovec are eligible, as well as those who have not received treatment before. Other eligibility criteria exist. 

For more information, please see below to consult the poster.

ResearchTreatmentsClinical trialsMay 31, 2022

Roche Community Update: FDA (U.S. Food and Drug Administration) approves a label expansion for risdiplam

Today, the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). This means that risdiplam is now approved in the US to treat SMA in children and adults of all ages.