Duchenne News

June 28, 2024
On June 28, 2024, Capricor Therapeutics announced additional positive 3-year safety and efficacy results from its ongoing HOPE-2 open label extension (OLE) study with its lead asset, deramiocel (CAP-1002), for...
June 24, 2024
DUVYZAT is a prescription medicine that is used for the treatment of Duchenne muscular dystrophy in people 6 years of age and older.
June 24, 2024
REGENXBIO shares a press release detailing updates on its Phase I/II AFFINITY DUCHENNE® trial.
June 20, 2024
Breaking news: US FDA has expanded approval of Sarepta Therapeutics’ gene therapy, Elevidys to Duchenne muscular dystrophy patients ages 4 and above. Elevidys has received traditional FDA approval for ambulatory...
June 4, 2024
Patients treated with CAP-1002 continue to show positive benefits after 3 years of treatment in both the PUL 2.0 and LVEF measures when compared to an external comparator dataset of...
May 20, 2024
On May 20, 2024, PTC Therapeutics announced that the European Commission (EC) has decided not to adopt the CHMP’s negative opinion of January 24, 2024 on the annual renewal of...
May 20, 2024
On Monday, May 20, Dyne Therapeutics announced positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 and its ongoing Phase...
May 10, 2024
REGENXBIO shares financial results for the first quarter ending March 31, 2024.
May 7, 2024
Tuesday, May 7, 2024 – We at Jett Foundation are deeply saddened to learn about the devastating loss of a child participating in Pfizer’s Phase 2 DAYLIGHT clinical trial for...
May 3, 2024
Pfizer shares that FDA has granted RMAT designation for gene therapy in development for Duchenne.

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