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November 11, 2019November 13, 2019

Clinical Trial Program Testing RG6206 in DMD Boys Has Been Discontinued

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The clinical program evaluating the safety, tolerability, and efficacy of RG6206 in boys with Duchenne muscular dystrophy (DMD) has been discontinued, according to a letter to the DMD community by ... Read more

November 9, 2019November 9, 2019

Local November 9th MDA Disease Education Events Span East, West Coast

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The Muscular Dystrophy Association’s (MDA) efforts to support and advocate for a wide range of neuromuscular diseases are felt by patient communities nationwide. While the MDA’s scope is indeed ... Read more

November 8, 2019November 8, 2019

Experimental Cell Therapy Improves Cardiac Function in Mouse Model of DMD, Study Says

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Treatment with a cell therapy candidate called dystrophin expressing chimeras (DECs) increased dystrophin levels in heart muscle and improved cardiac function in mice with Duchenne muscular dystrophy (DMD), a ... Read more

November 6, 2019November 6, 2019

Investigational Therapy Losmapimod Targets Root Cause of FSHD, Early Study Suggests

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The investigational oral treatment losmapimod is able to suppress the toxic activity of the DUX4 gene, the root cause of muscle degeneration in facioscapulohumeral muscular dystrophy (FSHD), new research ... Read more

November 4, 2019November 4, 2019

BIIB100 Improves Motor Function, Slows DMD Progression in Animal Models, Study Finds

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An experimental therapy called BIIB100 improved motor function and slowed the progression of Duchenne muscular dystrophy (DMD) in animal models of the disease, a study shows. These findings suggest ... Read more

November 1, 2019November 1, 2019

PPMD Grants Funding for Research on Cardiac Health in People with Duchenne

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Parent Project Muscular Dystrophy (PPMD) has awarded a total of $148,000 to researchers at Nationwide Children’s Hospital and the University of Missouri to help better understand and improve the ... Read more

October 30, 2019October 30, 2019

Translarna Preserves Lung Function, Muscle Strength in DMD Boys, Real-World Data Show

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Treatment with Translarna (ataluren) preserves lung function and muscle strength in boys with Duchenne muscular dystrophy (DMD), real-world data show. The most recent results from the ongoing STRIDE registry ... Read more

October 28, 2019October 28, 2019

Vamorolone Designated Promising Innovative Medicine in UK for DMD Treatment

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The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted promising innovative medicine (PIM) designation to ReveraGen’s vamorolone for the treatment of Duchenne muscular dystrophy (DMD). PIM designation indicates that ... Read more

October 25, 2019October 25, 2019

Novel Gene Therapy Safely Prevents Muscle Deterioration in Animal Models of DMD, Study Reports

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A novel one-time gene therapy that leads to the production of a dystrophin-related protein called utrophin prevents muscle deterioration and results in muscle regeneration in animal models of Duchenne ... Read more

October 23, 2019October 23, 2019

Vamorolone Extension Trial Supports Muscle Gains in DMD with Fewer Side Effects, Topline Data Show

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Vamorolone, an experimental treatment for Duchenne muscular dystrophy (DMD) being developed by ReveraGen Biopharma, continues to improve muscle function in DMD patients with fewer side effects than standard corticosteroids, topline ... Read more

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