Research Administration

MDA's 2015 Scientific Conference points the way toward tomorrow's therapies

posted on April 21, 2015 - 9:07am
Last month, MDA brought together more than 400 of the nation’s leading scientists, researchers and professionals specializing in neuromuscular disease research and care at its 2015 Scientific Conference in Washington, D.C., March 11–14. This pre-eminent gathering, hosted every other year, was designed to accelerate discoveries and drug development for families fighting neuromuscular diseases.

Research updates and clinical trials information

posted on January 5, 2015 - 9:07am
Quest Winter 2015
What About My Disease? Readers sometimes wonder what’s happening with research for their diseases when they don’t see news about them for a while in the pages of Quest. But keep in mind: Research that seems to be for one disorder often has spillover implications for others.
posted on October 3, 2013 - 9:15am
Quest Vol. 20, No. 4
In its Summer 2013 round of grants, MDA awarded 31 new grants totaling $8.5 million. 

A program that prepares young scientists for careers in drug development has awarded a grant for the study of muscle repair-boosting compounds

posted on August 22, 2013 - 5:00am
Christopher Penton recently completed his doctorate (Ph.D.) in integrated biomedical sciences at Ohio State University in Columbus.

30-person trial will test the safety, tolerability and pharmacokinetics of the experimental drug HT-100 in boys with Duchenne muscular dystrophy

posted on July 30, 2013 - 1:51pm
Update (Jan. 6, 2014): In a Dec. 23, 2013, announcement, Halo said the U.S. Food and Drug Administration (FDA) had placed this trial on "clinical hold" because of some adverse events that occurred in dogs being treated with HT-100. No further dosing of patients will occur until this issue has been resolved.

50 women are needed to fill out a questionnaire about the effects of myotonia congenita and paramyotonia congenita on pregnancy and delivery — and vice versa

posted on July 19, 2013 - 5:00am
Upate (Dec. 18, 2013): This study of pregnancy and delivery in women with myotonias is no longer recruiting new participants. The investigators will continue to collect data forms from people with whom they are already in contact. Investigators at the University of Rochester (N.Y.) are seeking participants for a questionnaire-based study of pregnancy and delivery in women with nondystrophic...

Targeting multiple pathways in mice with a disorder resembling merosin-deficient congenital muscular dystrophy shows more promise than aiming at one pathway at a time

posted on July 11, 2013 - 11:15am
Researchers at Boston University, supported in part by MDA, say their experimental two-pronged strategy for merosin-deficient congenital muscular dystrophy (MDC1A) was highly successful in a mouse model of this disease and should be further investigated as a potential treatment approach for patients.

Although the experimental Duchenne-Becker MD drug ataluren appeared to benefit walking ability in a clinical trial, some researchers question whether it works via stop codon read-through

posted on July 3, 2013 - 3:37pm
It's been widely accepted that the mechanism by which the experimental drug ataluren appears to benefit walking ability in Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD) is that it causes "read-through" of premature stop codons — genetic instructions that cause cells to stop making a protein before the process is complete.