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January 15, 2020January 15, 2020

Montana Rancher’s ‘Calves to Cure DMD’ Funds Duchenne Research

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Montana, the fourth-largest U.S. state in area, also is one of the least-populated — with maybe half a dozen Duchenne muscular dystrophy patients among its 1.1 million inhabitants. One ... Read more

January 13, 2020January 13, 2020

Solid Biosciences Cuts Workforce, Restructures to Focus on SGT-001 Development

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Solid Biosciences announced it will reduce its workforce by approximately one third to cut expenses and focus on advancing the development of SGT-001, its gene therapy candidate for the ... Read more

January 10, 2020January 10, 2020

Mount Everest Base Camp Trek Raises $60K for Duchenne Research

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Tonya Dreher wanted to do something dramatic on behalf of Hope for Gus, the New Hampshire-based foundation inspired by her son, Gus, 14, who has Duchenne muscular dystrophy (DMD). In ... Read more

January 8, 2020January 8, 2020

PPMD Funds Gene Editing Research to Improve Cardiac Health, Muscle Function in Duchenne

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Parent Project Muscular Dystrophy (PPMD) has granted $465,000 to Renzhi Han, PhD, a professor at the Ohio State University College of Medicine, to advance research into a targeted gene ... Read more

January 6, 2020January 6, 2020

Astellas, Audentes Merger May Lead to New Exon-skipping Treatments for MD

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Astellas Pharma recently agreed to acquire Audentes Therapeutics, a move it expects will result in faster development of potentially best-in-class therapies for rare neuromuscular diseases, including muscular dystrophy (MD). Audentes’ ... Read more

January 3, 2020January 7, 2020

Vyondys 53 Available to Duchenne Patients in the U.S.

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The newly approved therapy Vyondys 53 (golodirsen) is available for immediate distribution to treat people with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, according to the medicine’s developer Sarepta Therapeutics. ... Read more

December 19, 2019December 19, 2019

New IGNITE DMD Trial Data Promising for SGT-001, Company Working to Lift Clinical Hold

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Treatment with gene therapy candidate SGT-001 may lead to clinically meaningful improvements for patients with Duchenne muscular dystrophy (DMD), new data from two participants in the Phase 1/2 IGNITE ... Read more

December 18, 2019December 18, 2019

Potential DMD Treatment, ATL1102, Showing Safety and Muscle Strength Gain in Phase 2 Trial

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The experimental therapy ATL1102 is safe and continues to show evidence of improved upper muscle strength and function in all nine non-ambulatory boys with Duchenne muscular dystrophy (DMD) in ... Read more

December 17, 2019December 17, 2019

Wave Life Sciences Discontinues Development of Suvodirsen for DMD

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Following its failure to increase dystrophin levels of patients in an early-stage clinical trial, Wave Life Sciences has discontinued development of the investigational exon-skipping therapy suvodirsen for people with ... Read more

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Montana Rancher’s ‘Calves to Cure DMD’ Funds Duchenne Research

Solid Biosciences Cuts Workforce, Restructures to Focus on SGT-001 Development

Mount Everest Base Camp Trek Raises $60K for Duchenne Research

PPMD Funds Gene Editing Research to Improve Cardiac Health, Muscle Function in Duchenne

Astellas, Audentes Merger May Lead to New Exon-skipping Treatments for MD

Top 10 Muscular Dystrophy Stories of 2019

Vyondys 53 Available to Duchenne Patients in the U.S.

New IGNITE DMD Trial Data Promising for SGT-001, Company Working to Lift Clinical Hold

Potential DMD Treatment, ATL1102, Showing Safety and Muscle Strength Gain in Phase 2 Trial

Wave Life Sciences Discontinues Development of Suvodirsen for DMD

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