Yearlong, MDA-supported study comparing two prednisone dosing schedules in Duchenne MD found similar benefits and side effects
posted on July 20, 2011 - 9:52am
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.
Investigators are testing compounds designed to cause exon skipping in Duchenne MD and drugs that may increase blood flow in Duchenne and Becker MD
posted on June 21, 2011 - 9:08am
U.S. trial of eteplirsen for Duchenne MD delayed
Physicians and scientists will meet Jan. 21-22, 2011, in Columbus, Ohio, at an MDA-sponsored conference about cardiomyopathy in Duchenne muscular dystrophy
posted on January 18, 2011 - 4:06pm
Experts from around the world will gather Jan. 21-22, 2011, at an MDA-sponsored conference about the heart in Duchenne muscular dystrophy (DMD).
posted on January 1, 2011 - 12:47pm
R. Mark Payne, M.D., professor of pediatrics at Riley Heart Research Center, Indiana University, is a pediatric cardiologist whose medical specialty is intervention in children with heart disease, and who conducts research on heart disease due to mitochondrial defects.
In Friedreich’s ataxia (FA), heart problems are related to a deficiency of the frataxin protein, which is normally located in...
posted on January 1, 2011 - 12:33pm
Overall, idebenone has been shown to be safe and well-tolerated in a number of phase 1, 2 and 3 human clinical trials dating back to 1999. The most common side effects are mild to moderate gastrointestinal issues such as diarrhea, nausea and indigestion. Early trials at low doses first demonstrated the potential for idebenone to reduce FA-associated cardiomyopathy (heart muscle abnormality).
posted on October 1, 2010 - 12:25pm
Featured in this issue: First U.S. exon-skipping trial opens * Utah researchers seek families with Ullrich CMD or Bethlem myopathy * Acceleron receives Fast Track, Orphan Drug designations for ACE031 * BioMarin abandons BMN195 development
posted on June 30, 2010 - 4:41pm
If you are pregnant or plan to become pregnant, discuss with your physician any medications you are taking. In all cases where medication is required, the risk to the fetus must be weighed against the health of the mother.
