Can SonoThera’s RIPPLE platform redefine nonviral delivery in oversized genetic payloads?
Can SonoThera’s ultrasound platform reshape gene therapy delivery? Find out how its $125M Series B targets DMD, ADPKD, and XLAS with nonviral precision.
Is givinostat’s entry into Japan a turning point for DMD care and for JCR Pharmaceuticals’ rare disease strategy?
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 pivots into rare disease with FDA nod for (Z)-Endoxifen in Duchenne muscular dystrophy
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 […]
Could utrophin become the next frontier in DMD care? Inside Ractigen’s RNAa push
Ractigen Therapeutics has initiated human testing of RAG-18, a novel small activating RNA (saRNA) therapeutic candidate for Duchenne muscular dystrophy (DMD), by dosing the first patient in an investigator-initiated trial at Peking Union Medical College Hospital in Beijing. The study is being led by Professor Dai Yi, a recognized expert in neuromuscular disorders, and will […]