Drugs

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).

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).

In an Aug. 28, 2013, announcement, the Dutch biotechnology company announced favorable results for drisapersen and a new grant for continuing development of PRO045

posted on August 30, 2013 - 5:00am
Update (Oct. 15, 2013):  In an Oct. 15, 2013, webinar, Prosensa announced preliminary results for the phase 1-2 trial of PRO044, designed to target exon 44 of the dystrophin gene. Dystrophin production that is believed to have occurred in response to the drug was seen in 12 out of 21 muscle biopsies that could be evaluated. No drug-related serious adverse events have been reported.

Santhera Pharmaceuticals' investigational drug to treat respiratory dysfunction in Duchenne muscular dystrophy has shown benefit compared to a placebo in a phase 3 trial

posted on May 29, 2014 - 10:23am
Results from a phase 3, 65-participant trial of idebenone (brand names are Catena and Raxone) in boys with Duchenne muscular dystrophy (DMD) show the drug reduced the decline in respiratory function compared to a placebo, potentially paving the way toward regulatory approval.

A phase 2 clinical trial of rituximab in myasthenia gravis has now opened at 15 U.S. centers under the auspices of NIH's NeuroNEXT

posted on August 11, 2014 - 8:18am
Update (Sept. 15, 2015): As of Aug. 19, 2015, 30 participants have enrolled in the Rituximab in Myasthenia Gravis trial. The study currently is recruiting participants. For more information about the study or if you are interested in participating, please contact: MGstudy@yale.edu, or call 1-844-MGSTUDY (1-844-647-8839).

The experimental drug SMT C1100 is designed to increase utrophin production as a treatment for Duchenne MD

posted on April 24, 2012 - 4:00am

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
QUEST Vol. 15, No. 5
*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.”

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.