Posted by Michael Wonder on 03 Mar 2020
FDA grants rare paediatric disease designation to Stealth BioTherapeutics for elamipretide for the treatment of Barth syndrome
3 March 2020 - Stealth BioTherapeutics today announced that the U.S. FDA has granted rare paediatric disease designation for elamipretide for the treatment of Barth syndrome, an ultra-rare genetic condition.
Barth syndrome is an ultra-rare genetic condition characterised by cardiac abnormalities often leading to heart failure and reduced life expectancy, recurrent infections, muscle weakness and delayed growth.
