Muscular Dystrophies

DMD: Drisapersen Receives ‘Breakthrough Therapy’ Designation

Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.

BMD: Participants Sought for Registry Associated with Gene Transfer Trial

In connection with a gene transfer clinical trial, investigators at Nationwide Children's Hospital in Columbus, Ohio, are seeking people with Becker muscular dystrophy (BMD) for participation in a patient registry.

The investigators say the registry will provide information that will:

DMD: Eteplirsen Shows Continued Benefit at 84 Weeks

Eteplirsen, an experimental exon-skipping drug designed to treat Duchenne muscular dystrophy (DMD) caused by specific mutations in the dystrophin gene, continues to show sustained benefit on walking distance through week 84 of a phase 2b, 12-person study.

DMD Symptoms Show Up Early and Can Be Measured, Study Finds

Careful assessments of motor, cognitive and language function in very young children with Duchenne muscular dystrophy (DMD) show these children lag behind their age-matched peers well before obvious symptoms of the disease appear.

DMD: 'Permanent' Gene Repair Strategy Looks Good in Lab

Permanent repair of a faulty gene has long been a goal of researchers working to develop gene-based therapies. But many current gene modification strategies that have entered clinical trials have been based on temporary forms of gene correction — treatments that will need to be given frequently throughout a person's life.

MD Briefs: Registries Are Still Open

FSHD, MMD: Registry remains open

The University of Rochester Medical Center in New York state would like to remind people with facioscapulohumeral muscular dystrophy (FSHD) or myotonic muscular dystrophy (MMD, or DM), and their families, about its registry to advance research in these two disorders.

Moving Toward 'Next-Generation' Gene Therapy

Planning for the next generation of gene and stem cell therapies for muscular dystrophies — even as the first generation is still under development — was the theme of a joint symposium sponsored by MDA and the Association Française Contre les Myopathies (French Association Against Myopathies, or AFM) at the 16th annual meeting of the American Society of Gene & Cell Therapy (ASGCT).

DMD, BMD: Phase 3 Trial Opens for Stop Codon Read-Through Drug

A large-scale, multinational phase 3 trial of the experimental drug ataluren has opened its first trial site, in Cincinnati, Ohio.

UPDATE: ACE-031 Clinical Trials in Duchenne MD

Biopharmaceutical companies Acceleron Pharma and Shire announced April 21, 2011, that MDA-supported clinical trials of ACE-031 for Duchenne muscular dystrophy (DMD) have been halted.

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