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.

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.

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. 

The latest data from the FIREFISH study of risdiplam was presented at the 14th European Paediatric Neurology Society (EPNS) Congress.