Posted by Michael Wonder on 22 Oct 2024
Papillon Therapeutics receives rare paediatric disease designation from the US FDA for PPL-002 for the treatment of Danon disease
21 October 2024 - Papillon Therapeutics today announced that the US FDA has granted rare paediatric disease designation to Papillon’s PPL-002, an experimental treatment for Danon disease.
PPL-002 has previously received orphan drug designation.
