Duchenne Muscular Dystrophy (DMD)

Eteplirsen (AVI-4658) Boosts Dystrophin Production in DMD

Complete and extremely encouraging findings from a phase 1b-2 trial of eteplirsen (AVI-4658), an exon skipping drug in development to treat a portion of the Duchenne muscular dystrophy (DMD) population, show the compound is safe and well-tolerated, and that it can significantly increase production of the needed dystrophin protein in recipients without eliciting an unwanted immune response.

DMD: Daily, Weekly Prednisone Treatment Results About the Same

A one-year, MDA-supported study comparing a weekend-only prednisone treatment schedule with a daily prednisone schedule in boys with Duchenne muscular dystrophy (DMD) has found that the two treatment regimens provide about the same benefits and have approximately the same side-effect profile.

DMD Guidelines — Corticosteroids

In January 2005, the American Academy of Neurology (AAN) released its long-awaited report on the use of corticosteroids (prednisone or deflazacort) in Duchenne muscular dystrophy (DMD). The report is also published in the Jan. 11 issue of Neurology. It finds that corticosteroids are beneficial in DMD, but that some drawbacks to their use must be considered.

Last Updated: 
Sat, 01/01/2005 - 11:46

Milestones in DMD Research

Download a printable PDF of this entire booklet PDF

MDA has funded Duchenne muscular dystrophy research since 1950, and has paid for more research into this disease than any nongovernmental agency. It also provides the most comprehensive services program of any nonprofit organization in the country.

Last Updated: 
Thu, 01/01/2009 - 20:06

DMD/BMD Guidelines — Cardiovascular Care

In December, the Section on Cardiology and Cardiac Surgery of the American Academy of Pediatrics published its recommendations for cardiac care in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) and carriers of those dystrophies in its journal, Pediatrics.

Summary of the recommendations from the American Academy of Pediatrics:

Last Updated: 
Wed, 03/01/2006 - 13:40

DMD Guidelines — Anesthesia Care

MDA, in collaboration with the American College of Chest Physicians, has issued a consensus statement on management of patients with Duchenne muscular dystrophy (DMD) undergoing anesthesia or sedation.

The complete statement is published in the December 2007 issue of the journal Chest. A technical abstract and list of authors is available on the journal’s website. (The full paper requires a journal subscription.)

Last Updated: 
Tue, 01/01/2008 - 13:38

DMD Guidelines — Respiratory Care

The Aug. 15, 2004, issue of the American Journal of Respiratory and Critical Care Medicine, published by the American Thoracic Society (ATS), contains a set of guidelines about respiratory care in Duchenne muscular dystrophy (DMD) prepared by a special ATS working group. Members of this group are experts in DMD respiratory care, generally managing the respiratory care of patients being seen at Muscular Dystrophy Association (MDA) clinics.

Last Updated: 
Sun, 08/01/2004 - 13:29

Duchenne Muscular Dystrophy Care Guidelines

A comprehensive set of clinical care recommendations for the diagnosis and management of Duchenne muscular dystrophy (DMD) is now available to medical professionals and families.

The complete recommendations, in printable PDF format, can be read here:

Last Updated: 
Tue, 12/01/2009 - 13:26