Wave Life Sciences receives FDA rare paediatric disease designation for WVE-N531 for the treatment of Duchenne muscular dystrophy

Wave Life Sciences

12 August 2024 - Wave Life Sciences today announced that the US FDA has granted rare paediatric disease designation to WVE-N531 for the treatment of boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. 

WVE-N531 is currently being evaluated in the potentially registrational FORWARD-53 clinical trial and Wave expects to deliver data, including dystrophin protein expression from muscle biopsies after 24 weeks of treatment, in the third quarter of 2024.

Read Wave Life Sciences press release

Michael Wonder

Posted by:

Michael Wonder