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.
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.
In January 2005, the American Academy of Neurology (AAN) released its long-awaited report on the use of corticosteroids (prednisone or deflazacort) inDuchenne 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.
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.
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.)
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.