Array of treatments means myasthenia gravis usually isn't so grave
posted on May 1, 2003 - 9:47am
Charles Ranly, who's had MG for eight years, oversees the plantings at his Texas country club. Photo by Charlie Bublik
A decision on approval for Lumizyme, a treatment for Pompe disease, is expected in June
posted on March 19, 2010 - 9:25am
Biotechnology company Genzyme, of Cambridge, Mass., expects the U.S. Food and Drug Administration (FDA) to rule by June 17, 2010, on Genzyme's application to market its laboratory-developed enzyme Lumizyme (alglucosidase alfa) in the United States.
Lumizyme and its near-twin, Myozyme, replace the acid maltase enzyme deficient in people with Pompe disease (acid alpha-glucosidase deficiency or acid...
Once incorrectly diagnosed and improperly treated, they're now among the most treatable neuromuscular disorders
posted on June 1, 2001 - 12:05pm
When Phillip Martin was a baby, most people — even doctors — didn't notice anything unusual about him, except perhaps that he was a little quiet compared to other babies. But as they watched him grow, Richard and Daina Martin of Kelseyville, Calif., began to sense their son might have a physical disability.
Phillip couldn't hold his head up by himself until he was nearly 6 months old, Daina...
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).
The experimental drug SMT C1100 is designed to increase utrophin production as a treatment for Duchenne MD
posted on April 24, 2012 - 4:00am
The experimental drug SMT C1100, designed to treat Duchenne muscular dystrophy (DMD) by increasing production of the muscle protein utrophin, will move from laboratory testing to a trial in healthy human volunteers, according to its developer, Summit Corporation PLC.
MDA has awarded $1.5 million to ReveraGen BioPharma to develop a drug with the benefits of prednisone, but without the side effects, for Duchenne MD
posted on May 3, 2012 - 4:00am
The Muscular Dystrophy Association has awarded $1,549,725 to ReveraGen BioPharma, a Rockville, Md., biotechnology company, for development of a dissociative glucocorticoid to treat Duchenne muscular dystrophy (DMD).
The Food and Drug Administration has OK'd the first U.S. treatment specifically for late-onset Pompe disease
posted on May 25, 2010 - 12:34pm
The U.S. Food and Drug Administration (FDA) has approved U.S. sales of the enzyme-replacement drug Lumizyme, the first treatment in the U.S. specifically for late-onset Pompe disease.
Specialists, support groups and stimulant medications help families cope with the unusual cognitive and personality effects of type 1 myotonic dystrophy
posted on September 1, 2008 - 2:27pm
*Many people interviewed for this article asked not to be identified to protect the privacy of affected family members.
“Everybody knows the word apathy,” says a California woman whose 25-year-old daughter’s type 1 myotonic dystrophy (MMD1, sometimes called DM1) was diagnosed just a few years ago. “People use the word loosely. I don’t think it does justice to the reality of this disease.”
"Lumizyme has stopped the progression of my disease," says one of first to receive the drug since the FDA granted commercial approval
posted on June 21, 2010 - 4:25pm
Monique Griffin of Orlando, Fla., was one of the first in the nation to receive the commercially available treatment Lumizyme for acid maltase deficiency (AMD, or Pompe disease).
After a year of planning, new recommendations for physicians caring for DMD patients have been released
posted on December 3, 2009 - 4:23pm
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:
