Disease registries are seeking participants with CMD, FSHD, LGMD, MMD and Pompe disease
FSHD, MMD: Registry remains open
The University of Rochester Medical Center in New York state would like to remind people with facioscapulohumeral muscular dystrophy (FSHD) or myotonic muscular dystrophy (MMD, or DM), and their families, about its registry to advance research in these two disorders.
Longtime MDA grantee Michio Hirano, M.D., talks about the science of treating genetic disorders of energy production
On a Thursday afternoon in July 2012, Michio Hirano, a professor of neurology, was in his office at Columbia University Medical Center, high above Manhattan’s upper west side.
Featured in this issue: Duchenne muscular dystrophy exon skipping trial ** Idebenone in DMD ** Gene injections for type 2D limb girdle muscular dystrophy ** Patient registry for LGMD2B and Miyoshi myopathy
The following article contains items about: Friedreich's ataxia, Charcot-Marie-Tooth disease, myotonic muscular dystrophy type 1, amyotrophic lateral sclerosis, Emery-Dreifuss muscular dystrophy and distal muscular dystrophy (Miyoshi myopathy)
Closer look questions risks of medications for people with CMT
Worsening of Charcot-Marie-Tooth (CMT) disease as a result of medication use may not be as worrisome as popular belief has portrayed it, say investigators who recently culled data from medical literature and from 209 people in the CMT North American Database, an MDA-funded registry.