Improving Newborn Screening for Neuromuscular Disorders

MDA recognizes the importance of early diagnosis for neuromuscular disorders to optimize opportunities for effective treatment. This includes supporting initiatives to understand the impact of newborn screening in neuromuscular disorders. In early March, MDA sponsored an international conference in St. Louis, MO, “Newborn Screening for Neuromuscular Diseases: Improving Patient Outcomes and Evaluating Public Health Impact,” that . . .

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$3 Million NIH Grant Propels Vamorolone to Phase 2 Testing for Treatment of DMD

ReveraGen BioPharma has announced that it has received a $3 million grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health to conduct phase 2 clinical trials of vamorolone (previously VBP15) in boys with Duchenne muscular dystrophy (DMD). Vamorolone, a “dissociative steroid,” is an anti-inflammatory compound. Researchers hope . . .

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Meet Jeramiha, MDA’s 2016 State Goodwill Ambassador from West Virginia

Jeramiha Hometown: Weston, West Virginia Age: 14 Diagnosis: Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy. Favorite School Subject/Activities: I love anything to do with sports! Favorite People and/or Pets: I live with my mom Ashley, stepdad Daniel, and siblings Dakota, Jayleigh, and Malik. . . .

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Expanded Access Program Provides Deflazacort to U.S. Patients with DMD

Marathon Pharmaceuticals announced today that it is expanding patient access to deflazacort, its investigational therapy for Duchenne muscular dystrophy (DMD). Medical centers across the country are now participating in the open-label expanded access program (EAP) called ACCESS DMD to provide deflazacort to qualified U.S. patients at no charge. Deflazacort, a corticosteroid, works as an anti-inflammatory . . .

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PTC Therapeutics Remains Committed to Getting Translarna Approved for DMD in the United States

In two webcasts held Feb. 29 — one for investors and one for Duchenne muscular dystrophy (DMD) patients and families — PTC Therapeutics addressed the Refuse to File letter it received last week from the U.S. Food and Drug Administration (FDA) stating that PTC’s New Drug Application (NDA) for Translarna (ataluren) to treat DMD was not sufficient . . .

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FDA Extends Decision Deadline Date for Eteplirsen to May 26, 2016

Background: Sarepta Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has extended the decision goal date for eteplirsen by a standard extension period of three months. The new date by which the FDA must make a decision about whether to approve eteplirsen for the treatment of Duchenne muscular dystrophy (DMD) is May 26, 2016. . . .

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FDA Issues Response to BioMarin’s DMD Treatment

Press Release: FDA Issues Complete Response Letter for KyndrisaTM for Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping Summary: BioMarin reported today that it has received a Complete Response Letter from the U.S. Food and Drug Administration (FDA) indicating that the review cycle for the company’s New Drug Application for drisapersen (Kyndrisa) to treat DMD is . . .

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Eli Lilly Provides Update on Phase 3 Tadalafil Trial for DMD

Please read the message below from Eli Lilly regarding the results of their phase 3 trial in Duchenne muscular dystrophy (DMD): Update on Phase 3 Tadalafil Trial We are writing to share disappointing news about our Phase 3 study of tadalafil in approximately 330 patients with Duchenne muscular dystrophy (DMD). We recently completed analysis of . . .

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Muscular Dystrophy Association to Collaborate with Catabasis Pharmaceuticals on DMD Drug

CAMBRIDGE, MA, and CHICAGO, IL, February 5, 2016 – Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage drug development company built on a pathway pharmacology technology platform, and the Muscular Dystrophy Association (MDA), today announced a collaboration to support the Part B portion of the MoveDMD clinical trial of CAT-1004, a novel product candidate for the treatment . . .

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