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March 18, 2020March 18, 2020

Capricor Therapeutics Expands Exosome Technology for DMD

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Capricor Therapeutics is expanding the development of exosome-based technologies that are being tested as a potential way to treat Duchenne muscular dystrophy (DMD). Capricor’s lead candidate for DMD is CAP-1002, ... Read more

March 17, 2020March 17, 2020

AdipoRon Eases Inflammation, Improves Motor Function in DMD Mice, Study Shows

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Oral treatment with AdipoRon reduced inflammation in muscle, promoted muscle fiber proliferation, and improved muscle and motor function in a mouse model of Duchenne muscular dystrophy (DMD), a study ... Read more

March 16, 2020March 16, 2020

Vyondys 53 Boosts Dystrophin Production in Boys With DMD, Early Results Show

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Treatment with Vyondys 53 (golodirsen) for 48 weeks increases levels of dystrophin protein by 16-fold in boys with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, updated results of a Phase ... Read more

March 13, 2020March 13, 2020

MDA Let’s Play Uses Gaming to Raise Funds for Neuromuscular Disorders

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The Muscular Dystrophy Association (MDA) is launching MDA Let’s Play, a platform to raise funding and awareness about muscular dystrophy and related neuromuscular disorders through gaming. “We’ve always been an iconic ... Read more

March 12, 2020March 12, 2020

Increlex Improves Growth, But Not Muscle Function in DMD, Early Trial Shows

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Treatment for six months with Increlex (mecasermin), an injectable form of insulin-like growth factor-I (IGF-I), enhanced growth in boys with Duchenne muscular dystrophy (DMD), a Phase 1/2 trial shows. ... Read more

March 11, 2020March 11, 2020

‘Napa in Newport’ Raises More Than $1M for CureDuchenne

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The sixth annual Napa in Newport wine auction raised more than $1 million to support CureDuchenne’s mission to extend and enhance the lives of people with Duchenne muscular dystrophy ... Read more

March 10, 2020March 10, 2020

Pediatric Rare Disease Therapies Increase, But Most Repurposed, Study Finds

Muscular Dystrophy News, News

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more

March 10, 2020March 10, 2020

Clinical Trial into Natural History of LGMD2i Enrolling in US, Denmark

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A new clinical trial is seeking about 80 people with limb-girdle muscular dystrophy type 2i (LGMD2i) to gather information on this disease’s progression that may help in advancing potential ... Read more

March 9, 2020March 9, 2020

PerkinElmer Launches New Genetic Test for FSHD Type 1

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PerkinElmer Genomics has launched a new test that is able to accurately detect the shortening of the D4Z4 gene region which causes facioscapulohumeral muscular dystrophy (FSHD) type 1. FSHD ... Read more

March 6, 2020March 23, 2020

L-arginine in Diet May Improve DMD Gut Function, Mouse Study Suggests

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Supplementing the diet with the amino acid L-arginine may improve gut function in people with Duchene Muscular Dystrophy (DMD), a study in mice suggests. The study, “Spatiotemporal Mapping Reveals ... Read more

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