A program that prepares young scientists for careers in drug development has awarded a grant for the study of muscle repair-boosting compounds
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
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
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
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
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.
The FDA will speed up the review process for a promising exon-skipping drug designed to treat Duchenne MD caused by specific dystrophin mutations
Multinational pharmaceutical company GlaxoSmithKline (GSK) has announced that its experimental Duchenne muscular dystrophy (DMD) drug drisapersen has received breakthrough therapy designation from the U.S. Food and Drug Administration.
Insights from pediatric neurologist Carsten Bönnemann
Not long ago, a diagnosis of congenital muscular dystrophy (CMD) in a baby could not provide much information or comfort for a parent.
It generally meant the child was very weak — often described as “floppy” — at birth, and may have had difficulty breathing or swallowing. The diagnosis, as well as the prognosis, was uncertain, and treatment was almost nonexistent.