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282,745 Results for "dmd therapeutics inc.rss".
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Duchenne muscular dystrophy
Sarepta’s DMD Gene Therapy Staves Off Disease Three Years After Treatment
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.
January 26, 2026
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2 min read
·
Dan Samorodnitsky
Press Releases
Dyne Therapeutics Announces Positive Topline Results from Phase 1/2 DELIVER Trial of Z-Rostudirsen in Duchenne Muscular Dystrophy (DMD)
December 8, 2025
·
15 min read
Duchenne muscular dystrophy
Capricor Heads Back to FDA With Pivotal Results for DMD Cardiomyopathy Therapy
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.
December 3, 2025
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3 min read
·
Heather McKenzie
Duchenne muscular dystrophy
Dyne’s Exon Skipper Delivers ‘Best Ever’ Functional Outcomes in Early DMD Study
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.
December 8, 2025
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2 min read
·
Tristan Manalac
Press Releases
Precision BioSciences Sets Strategic Priorities for 2026 Focused on Continued Advancement of Clinical-Stage Programs PBGENE-HBV for Chronic Hepatitis B and PBGENE-DMD for Duchenne Muscular Dystrophy
January 12, 2026
·
11 min read
Duchenne muscular dystrophy
Sarepta’s DMD Exon-Skippers Fail Confirmatory Study, Stock Craters
Sarepta nevertheless plans to push for full FDA approval of Vyondys 53 and Amondys 45 based on what it said are “encouraging trends” in efficacy.
November 4, 2025
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2 min read
·
Tristan Manalac
Duchenne muscular dystrophy
With Prasad Back at FDA, Capricor To Fight DMD Cardiomyopathy Rejection
Capricor Therapeutics met with the FDA last week for a type A meeting, during which CEO Linda Marbán aimed to explain to the regulator that it got it wrong. Capricor plans to resubmit the application based on deramiocel’s existing dataset.
August 20, 2025
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7 min read
·
Heather McKenzie
Press Releases
Avidity Biosciences Announces U.S. Managed Access Program (MAP) for Investigational Therapy del-zota in DMD44
November 20, 2025
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7 min read
Press Releases
Dyne Therapeutics Announces FDA Breakthrough Therapy Designation for DYNE-251 in Duchenne Muscular Dystrophy (DMD)
August 5, 2025
·
8 min read
Press Releases
Five-year data of AGAMREE® (vamorolone) in patients with DMD show improved safety profile with comparable effectiveness to standard of care corticosteroids
November 4, 2025
·
7 min read
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