This report on treatment development for Duchenne muscular dystrophy contains news about fighting fibrosis (scarring), treating DMD-related heart disease, exon skipping and the corticosteroid deflazacort

posted on February 2, 2015 - 12:04pm
Development of treatments for Duchenne muscular dystrophy (DMD) continues to advance. Many of the new investigational drugs are potentially applicable to all DMD patients, while a few target those with specific mutations in the dystrophin gene. Fighting fibrosis

A new MDA grant will defray travel costs for participants in a large-scale trial to find the optimal corticosteroid regimen for boys with Duchenne MD

posted on October 24, 2013 - 2:00pm
Robert Griggs, a professor of neurology at the University of Rochester (N.Y.), has received an MDA grant of $237,316 over three years to support travel costs for North American participants in a large, multinational trial to determine which corticosteroid treatment regimen is best for children with Duchenne muscular dystrophy (DMD).
posted on October 3, 2013 - 9:05am
Quest Vol. 20, No. 4
In kindergarten, Mo Gerhardt played tee ball and soccer, and while his hand-eye coordination was excellent, his strength wasn’t. Matthew (“Mo”) Gerhardt of Bath, Mich., will be 36 years old this fall.

MDA grant will support a study of prednisone in 25 boys with Duchenne muscular dystrophy who are less than 2½ years old

posted on August 20, 2013 - 5:00am
Pediatric neurologist Anne Connolly has received an MDA grant to study the effects of prednisone on Duchenne muscular dystrophy in very young children. MDA has awarded a grant to Anne Connolly, a pediatric neurologist at Washington University in St.

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.

A report on drug development in neuromuscular diseases as of January 2009

posted on January 1, 2009 - 2:37pm
QUEST Vol. 16, No. 1
In the era of molecular biology, the drug development process has moved from a “let’s try it and see what happens” approach to a scientifically based process of discovery and application. For many of the diseases in MDA’s program, drug discovery begins with gene discovery — identifying a gene that, when flawed, causes a disease.

Corticosteroids are useful treatments for some neuromuscular diseases, but finding the right medication and dosing schedule isn't always smooth sailing

posted on May 7, 2007 - 1:58pm
QUEST Vol. 14, No. 3
Corticosteroids (also known as glucocorticoids) that are commonly used to treat neuromuscular disorders include prednisone, prednisolone and deflazacort. All are powerful medications that suppress the immune system and decrease inflammation.
posted on May 1, 2007 - 2:09pm
QUEST Vol. 14, No. 3
Date Trial or Study Benefits Side Effects 1970s-1980s 4 trials used prednisone in high doses ranging from 1.5 mg/kg/day to 5 mg/kg on alternate days prolongation of walking by about 2 years in one study; one study showed no benefit