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With a clutch of key data and planned regulatory applications this year from Avidity Biosciences, REGENXBIO and Capricor Therapeutics, CureDuchenne CSO Michael Kelly sees “momentum” in the Duchenne muscular dystrophy pipeline, as Sarepta’s Elevidys leaves the door open.

· RIPPLE™ (Remote Induction of Pulsed Pressure Lateral to Energy) is a proprietary ultrasound-mediated delivery technology. · RIPPLE™ has been developed to enable efficient, redosable, targeted, and safe delivery of a diverse range of genetic medicines with broad tissue biodistribution, including skeletal, cardiac, and diaphragm muscles.

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.

Dyne’s exon-skipping therapy zeleciment rostudirsen resulted in an approximately sevenfold increase in dystrophin levels at six months and elicited functional improvements that are the “best ever” for this treatment class, Stifel analysts said.

Six months after receiving a surprise rejection due to what the FDA called “lack of substantial evidence of effectiveness,” Capricor’s cell therapy deramiocel showed significant benefits in upper-limb function and slowed decline in cardiac function in a Phase III trial.