CureDuchenne welcomes the recent initiative the Food and Drug Administration (FDA) has taken to improve efficiencies in drug development, manufacturing, and the review process for new drug applications that incorporate […]
See the previous update from June 22, 2023 here FDA Grants Traditional Approval for Ambulatory Individuals and Accelerated Approval for Non-Ambulatory Individuals to Sarepta Therapeutics’ ELEVIDYS Gene Therapy for Duchenne […]
We are blessed that our son Momo joined our family through adoption. It was a long road to bring him home that led us to understand that God’s plans are […]
My grandson Shepherd has been living with his Duchenne diagnosis for 5 years. Since that day in 2019, our family has turned into crusaders for the Cause. It has been […]
Our Duchenne journey began like so many others. Gabe, our fourth son, wasn’t reaching his developmental milestones. His doctor told us there was nothing wrong with him, and that, as the […]
Our son Grant is six years old and was diagnosed with Duchenne Muscular Dystrophy at age three. Duchenne is a genetic disease that destroys muscle, all of them. We are […]
PTC Therapeutics Provides Key Regulatory Updates
REGENXBIO ANNOUNCES NEW POSITIVE INITIAL EFFICACY DATA FROM AFFINITY DUCHENNE® TRIAL
Santhera Receives Approval for AGAMREE® (Vamorolone) as a Treatment for DMD in the UK.
