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News & Resources

GsMTx4 a Promising ‘Out-of-the-Box’ Therapy for Advanced DMD, Mouse Study Shows

A new investigational therapy called GsMTx4, developed by researchers at the University of Buffalo and based on a molecule found in tarantula venom, is able to prevent the loss of muscle mass and muscle injury in a mouse model of advanced Duchenne muscular dystrophy (DMD). These findings were published in the study, “GsMTx4-D provides protection to the D2.mdx mouse,” […]


PTC Therapeutics Announces Initial Data from Patient Registry

PTC Therapeutics Announces Initial Data from Patient Registry Demonstrating Translarna™ (ataluren) Slows Disease Progression in Children with Duchenne Caused by a Nonsense Mutation SOUTH PLAINFIELD, N.J., Oct. 6, 2018 /PRNewswire/ — PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced preliminary data from the first international drug registry for Duchenne patients receiving Translarna™ (ataluren), underscoring the long-term clinical benefit of Translarna when […]


Sarepta Gene Therapy Appears To Help Fourth Boy With Duchenne Muscular Dystrophy

An experimental gene therapy dramatically improved the ability of a young boy with Duchenne muscular dystrophy to rise from the ground and climb stairs, repeating and improving on results seen in three previous patients who received the treatment, which is being developed by Sarepta Therapeutics of Cambridge, Massachusetts. “The cautionary note is this all has […]


Reclaim the Day

We’re pleased to share this message from our friends at Stand for Duchenne about a new initiative they have created called Reclaim the Day. Many Duchenne families, including ours, will never forget the day their children were diagnosed. The feeling of shock, panic, and helplessness is only a fraction of the emotions that we all […]


Catabasis Pharmaceuticals Initiates Phase 3 PolarisDMD Clinical Trial For Edasalonexent In Duchenne Muscular Dystrophy

– Pivotal Study of Novel Inhibitor of NF-kB, a Key Driver of Skeletal Muscle Disease and Cardiomyopathy in Duchenne — CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the initiation of PolarisDMD, the Company’s Phase 3 trial for edasalonexent in Duchenne muscular dystrophy (DMD). Edasalonexent inhibits NF-kB, which is the key link […]


Sarepta’s Duchenne MD trial back on schedule

Sarepta’s early-stage clinical trial for a gene therapy for Duchenne muscular dystrophy is back on schedule, the Cambridge biotech said Monday. The Food and Drug Administration had put the trial on hold in July. The FDA ordered the hold after small fragments of DNA turned up in the treatment, which is being used at Nationwide […]


Pull a Plane to Defeat Duchenne

On Saturday, September 22 more than 100 people came out to the Jet Aircraft Museum in London, Ontario for the first “Pull a Plane to Defeat Duchenne”. This first-time event was held in partnership with Western University’s Commercial Aviation Management program. Participants teamed up to pull a 10,000lb, beautifully-restored Canadian T33 jet 50 metres across the […]


Potential DMD Therapy Vamorolone Shows Positive Effects in Phase 2a Trial, Study Reports

Vamorolone, a Duchenne muscular dystrophy (DMD) treatment candidate, showed anti-inflammatory activity and an improved safety profile over the glucocorticoid prednisolone in a Phase 2a clinical trial for boys 4 to 6 years old. Findings from the trial were published in the study, “Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory […]


Sarepta Receives Negative CHMP Re-examination Opinion for Eteplirsen

— Relying upon Scientific Advisory Group input, Sarepta will seek further scientific advice from European Medicines Agency on a possible path to bring eteplirsen to patients in Europe — CAMBRIDGE, Mass., September 21, 2018 (GLOBE NEWSWIRE) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial-stage biopharmaceutical company focused on the discovery and development of precision genetic […]


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