@FDA_KyleD@FDACDERDirector@SecKennedy@POTUS@RonJohnsonWI@SenRonJohnson Please help us and signal to @PTCBio that FDA will weigh #RealWorldEvidence and apply consistent standards across CDER/CBER when reviewing #Ataluren for stop-codon #Duchenne. Real-world outcomes truly matter. Our sons with a nonsense mutation are not eligible for exon-skipping; my son has been in Ataluren trials/extension since 2006 with no side effects. He lost ambulation at 22 (2015) and, at 32, has avoided typical pulmonary decline with 97% pulmonary scores. 90 day supply recieved 6/1. March 2026 we were notified that there was only a 6 month suppY. Clock is ticking. #RareDisease families can't risk losing time/abilities that can’t be regained. #TimeIsMuscle
@FDA_KyleD@FDACDERDirector@SecKennedy@POTUS@RonJohnsonWI@SenRonJohnson Please help us and signal to @PTCBio that FDA will weigh #RealWorldEvidence and apply consistent standards across CDER/CBER when reviewing #Ataluren for stop-codon #Duchenne. Real-world outcomes truly matter: a 13-year-old running; a 34-year-old maintaining breathing function. Our sons with a nonsense mutation are not eligible for exon-skipping; my son has been in Ataluren trials/extension since 2006 with no side effects. He lost ambulation at 22 (2015) and, at 32, has avoided typical pulmonary decline with 97% pulmonary scores. #RareDisease families can't risk losing time/abilities that can’t be regained. #TimeIsMuscle