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December 16, 2019December 16, 2019

FDA Authorizes Use of Test to Aid Newborn Screening for Duchenne MD

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The U.S. Food and Drug Administration (FDA) has authorized use of the first test intended to facilitate newborn screening for Duchenne muscular dystrophy (DMD). “Diagnostics that can safely and ... Read more

December 13, 2019December 13, 2019

FDA, in Reversal, Approves Vyondys 53 to Treat Duchenne Patients with Exon 53 Mutations

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The U.S. Food and Drug Administration has conditionally approved Vyondys 53 (golodirsen) to treat Duchenne muscular dystrophy (DMD) patients amenable to exon 53 skipping. This accelerated approval comes less than ... Read more

December 11, 2019December 11, 2019

GPM-owned Convenience Stores Run Holiday Campaign to Support Muscular Dystrophy Association

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GPM Investments, the sixth largest convenience store chain in the U.S., is running its “Tis the Season for Giving” holiday campaign throughout December to support the Muscular Dystrophy Association ... Read more

December 10, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 9, 2019December 9, 2019

Buffalo Bills Team with Med Students to Help MDA and Honor Duchenne Volunteer

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Two University at Buffalo (UB) medical school students were so moved by a classroom video presentation by a young man with Duchenne muscular dystrophy that they are working to ... Read more

December 6, 2019January 15, 2020

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

December 6, 2019January 15, 2020

Mitochondrial Protein’s Loss Linked to Muscle Weakness and Poor Repair in Early Study

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Loss of MICU1, a key regulator of calcium balance in cells, was seen to affect muscle function and repair in a study in mice and cells from people lacking ... Read more

December 4, 2019December 4, 2019

Avidity Secures $100M Funding to Advance Treatments for Myotonic Dystrophy, DMD

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Avidity Biosciences has obtained $100 million to advance its programs for muscle disorder therapies, including a potential Antibody Oligonucleotide Conjugates (AOC) treatment for myotonic dystrophy type 1 (MD1). Based on ... Read more

December 2, 2019December 2, 2019

New DMD Mouse Model Helps Monitor Real-Time Dystrophin Production, Study Reports

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A new mouse model of Duchenne muscular dystrophy (DMD) can help researchers test potential gene editing therapies by showing real-time levels of the dystrophin protein in muscles and the ... Read more

November 27, 2019November 27, 2019

Experts Barry Byrne, Jerry Mendell Lead NORD Webinar on Gene Therapy

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A glance around the walls of Barry J. Byrne’s office reveals a lot about the pediatric cardiologist who runs the Powell Gene Therapy Center at University of Florida (UF). In ... Read more

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