The FDA will make a decision whether or not to grant full approval of Sarepta’s gene therapy, Elevidys, by June 21,2024.
Fat embolism syndrome (FES) is a potentially life-threatening condition that can be triggered by bone fractures.
Finding the Right One: Advice from Experts on Dating with a Disability
REGENXBIO Announces Completion of Enrollment in Cohort 2 and Additional Positive Interim Data in AFFINITY DUCHENNE® Trial
By Jacob Gapko Jacob Gapko is 46 years old and has Duchenne muscular dystrophy. He uses a power wheelchair and non-invasive ventilation 24/7. He has a B.S. in physics and minors […]
Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51. In part B of the Phase 2 MOMENTURM study, ambulatory and […]
This year, our CureDuchenne blogs ran the gamut from FDA approvals to overcoming adversity to books and movies that represent people with disabilities to how to stay happy and healthy. […]
Books are a gateway for us to unlock our imaginations. To see ourselves in the characters that captivate our attention. To let our mind wander to places that wouldn’t have […]
Mother of two boys with Duchenne shares how she’s successfully advocated for her children with a rare disease as a mama bear.