Sarepta’s ELEVIDYS Cohort 8 targets non-ambulatory Duchenne patients with a sirolimus regimen. What the trial design reveals about the path to label restoration.
PTC Therapeutics has withdrawn its U.S. filing for Translarna. Find out what this means for Duchenne drug development and FDA expectations.
Sarepta’s gene therapy ELEVIDYS shows 3-year durability in Duchenne muscular dystrophy. Find out what it means for patients, payers, and global rollout.
Genethon advances gene therapies for Duchenne and Pompe disease. Find out what its nonprofit model and AskBio licensing deal mean for rare disease care.
Sarepta’s EMBARK 3-year readout could clarify ELEVIDYS’ long-term role in Duchenne care. Find out what’s at stake in this pivotal gene therapy trial.
Atossa Therapeutics Inc. has received Orphan Drug Designation from the U.S. Food and Drug Administration for its investigational compound (Z)-Endoxifen in the treatment of Duchenne muscular dystrophy (DMD). The designation, disclosed on January 16, 2026, supplements the previously granted Rare Pediatric Disease Designation for the same compound and indication. The move marks a notable regulatory […]
Swissmedic approves Santhera’s AGAMREE for Duchenne muscular dystrophy. Find out how vamorolone could reshape long-term steroid treatment strategies.
Can SonoThera’s ultrasound platform reshape gene therapy delivery? Find out how its $125M Series B targets DMD, ADPKD, and XLAS with nonviral precision.
Find out how Italfarmaco and JCR are bringing the DMD drug givinostat to Japan and what it means for rare disease innovation in Asia.
Atossa Therapeutics has received Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration for (Z)-Endoxifen in Duchenne muscular dystrophy (DMD), a strategic shift that positions the estrogen receptor modulator for possible use in pediatric neuromuscular disorders. If approved, the program could qualify for a transferable Priority Review Voucher, but the timeline and […]