posted on January 1, 2012 - 12:10pm
Researchers studying ‘natural history’ of four CMT subtypes
Participants are sought for a large-scale study, supported in part by MDA, to determine the natural history (general disease course) of four subtypes of Charcot-Marie-Tooth disease (CMT), with particular emphasis on correlations between genetic mutations and symptoms. The four subtypes are CMT1B, CMT2A, CMT4A and CMT4C.
posted on October 1, 2011 - 10:09am
Featured in this update:
Families, experts meet at BMD ConferenceThree trials study blood-vessel-dilating drugs in BMD, DMDResults of daily, weekly prednisone treatment about the same in DMD
posted on July 1, 2011 - 1:01pm
New CMS-causing genetic mutations indentified
Scientists supported in part by MDA have identified mutations in the gene for the plectin protein as a rare cause of a congenital myasthenic syndrome (CMS).
posted on March 31, 2011 - 10:04am
In this issue: MDA awards 44 new research grants * A recently discovered gene variant may indicate severity of DMD * Heart care in Duchenne MD and Becker MD addressed at MDA-sponsored meeting * Duchenne-Becker 'read-through' drug to begin development * Flow charts may aid in diagnosing CMT
posted on January 1, 2011 - 1:44pm
Sildenafil helps heart in mice lacking dystrophin
posted on October 1, 2010 - 12:15pm
Improved Gene Repair Strategy Helps Cells Fix DNA Errors in DMD
A new generation of molecules can help cells permanently repair errors in the dystrophin gene, fixing the underlying cause of Duchenne muscular dystrophy (DMD) — and potentially fixing the underlying genetic causes of other neuromuscular diseases as well, researchers report.
News on Duchenne muscular dystrophy, Friedreich's ataxia, autoimmune diseases, and cell and gene therapies
posted on September 24, 2010 - 1:39pm
Duchenne muscular dystrophy
