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.