Muscular Dystrophy News

March 10, 2020March 10, 2020

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

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

June 9, 2017June 9, 2017

Gene Therapy Shows Long–Lasting Effects in Animal Model of Myotubular Myopathy

Muscular Dystrophy News, News

A gene therapy treatment may restore the genetic mutations of myotubular myopathy, a type of muscular dystrophy that affects the skeletal muscles. In a previous study, a team of ... Read more

March 13, 2017

MDA Awards $7M in Grants for 29 Disease-Targeting Research Projects

Muscular Dystrophy News

The Muscular Dystrophy Association (MDA) has awarded $7 million in grants to 29 top researchers for projects it says will potentially make a big impact on muscular dystrophy and other life-threatening ... Read more

September 19, 2016September 19, 2016

BREAKING NEWS: FDA Approves Sarepta’s Exondys 51 (Eteplirsen) as First Therapy for Duchenne MD

Muscular Dystrophy News, News

The U.S. Food and Drug Administration has approved Sarepta Therapeutics’ therapy Exondys 51 (eteplirsen) as a treatment for Duchenne muscular dystrophy patients with a confirmed mutation of the dystrophin gene amenable ... Read more

June 2, 2016June 2, 2016

BioMarin Withdraws MAA for Kyndrisa (Drisapersen) in Europe

Muscular Dystrophy News, News

BioMarin Pharmaceutical Inc. has announced the withdrawal of its Marketing Authorization Application (MAA) from the European Medicines Agency (EMA) for Kyndrisa (drisapersen), a drug developed for Duchenne muscular dystrophy (DMD) amenable ... Read more

April 25, 2016April 26, 2016

UPDATED: FDA Panel Casts Some Doubt on DMD Drug Eteplirsen’s Accelerated Approval

Muscular Dystrophy News, News

UPDATE: Since this story was filed, Reuters news service is reporting that even more quickly than we had anticipated was likely, the expert panel convened by the FDA this ... Read more

April 1, 2016April 1, 2016

Marathon Pharmaceuticals Study May Support Deflazacort Approval For DMD

Muscular Dystrophy News, News

The glucocorticoid deflazacort might shortly be approved for treatment of Duchenne muscular dystrophy (DMD) in the US. In preparation for the approval process, Marathon Pharmaceuticals have released new data ... Read more

March 17, 2016May 20, 2016

New Biomarkers for Utrophin Protein Levels Likely to Aid DMD Therapy Now in Clinical Testing

Muscular Dystrophy News, News

Summit Therapeutics plc announced the publication of a study into new imaging techniques that appear to reliably and reproducibly measure utrophin protein levels and muscle fiber regeneration in muscle biopsies in Duchenne ... Read more

January 21, 2016

FDA Postpones Advisory Meeting on Potential DMD Therapy Due to Severe Winter Storm

Muscular Dystrophy News, News

Sarepta Therapeutics, Inc., announced that a U.S. Food and Drug Administration (FDA) advisory committee has been forced to postpone Friday’s scheduled hearing to review the company’s New Drug Application ... Read more

December 4, 2015December 3, 2015

Duchenne MD Patients May Receive Investigational Drug at No Cost

Muscular Dystrophy News, News

Marathon Pharmaceuticals has announced the launch of ACCESS DMD, its expanded access program (EAP) to provide deflazacort to qualified patients with Duchenne muscular dystrophy (DMD) under the authorization of the ... Read more