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February 15, 2019February 15, 2019

Golodirsen, Potential Exon 53 Skipping Therapy for Duchenne, Under Priority Review by FDA

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Sarepta Therapeutics announced that the U.S Food and Drug Administration (FDA) has accepted its application for a priority review of golodirsen (SRP-4053), a potential treatment for Duchenne muscular dystrophy (DMD) patients amenable ... Read more

February 13, 2019February 13, 2019

Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

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Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run ... Read more

February 13, 2019February 13, 2019

Parent Project Muscular Dystrophy Takes Its ‘End Duchenne Tour’ on Road Again for 2019

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With the goal of connecting with every U.S. family facing a Duchenne muscular dystrophy (DMD) diagnosis, Parent Project Muscular Dystrophy (PPMD) continues its multi-year community effort called the End ... Read more

February 11, 2019February 11, 2019

Duchenne Patients Again Being Treated in HOPE-2 Trial of Potential Cell Therapy, Capricor Announces

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Capricor Therapeutics announced that patient dosing has resumed in its HOPE-2 trial of CAP-1002, an investigative cell therapy for Duchenne muscular dystrophy The company placed a voluntary on dosing after ... Read more

February 8, 2019February 14, 2019

Defying the Odds of Living with Duchenne, Decade After Decade

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When it comes to Duchenne muscular dystrophy (DMD), Leonardo Feder knows he’s among the lucky ones. A resident of São Paulo, Brazil’s largest city, Feder was diagnosed with Duchenne ... Read more

February 6, 2019February 14, 2019

PPMD Funds Gene Therapy Research Aimed at Improving Cardiac Function in DMD Patients

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Parent Project Muscular Dystrophy (PPMD) has granted $105,000 to the University of Missouri School of Medicine to advance research into a mini-dystrophin gene therapy that may protect cardiac function in ... Read more

February 5, 2019February 6, 2019

ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration

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G71.01 is, literally, the code for Duchenne muscular dystrophy. Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and treatment-resistant epilepsies, which groups like Orphanet and the American Epilepsy Society ... Read more

February 4, 2019February 7, 2019

DMD Increases Susceptibility to Rare, Aggressive Muscle Cancer, Mouse Study Suggests

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Severe Duchenne muscular dystrophy (DMD) is linked to increased susceptibility to a rare and aggressive type of muscle cancer called rhabdomyosarcoma, a mouse study suggests. The finding was reported ... Read more

February 1, 2019February 6, 2019

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

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The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal ... Read more

February 1, 2019February 7, 2019

Popeyes Group Launches 15th Annual Campaign to Help MDA

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Popeyes Louisiana Kitchen’s franchisee Sailormen has launched the 15th Annual “Appetite for a Cure” campaign to enhance the lives of those living with muscular dystrophy (MD) and related disorders. Money ... Read more

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