A follistatin gene therapy compound gains orphan drug status; potential improvements are reported for utrophin-based therapies, exon skipping and stem cell transplantation
Update (Jan. 23, 2013): The "Building better utrophin" section was updated to reflect the availability of a Jan. 22, 2013, press release from the University of Missouri.
Below is a wrap-up of recent research news about the development of therapies for Duchenne, Becker and limb-girdle muscular dystrophies.
The drug improved heart function and survival in mice with lamin A/C mutations, which cause lamin A/C-related Emery-Dreifuss MD and type 1B limb-girdle MD
Update (June 1, 2012): This story was updated to include the availability of a podcast on this topic.
Top researchers are discussing notable developments in neuroscience, including research in many neuromuscular diseases, at the annual American Academy of Neurology meeting April 21-28
Top scientists and clinicians from around the world are discussing the latest research in neuroscience, and the care of individuals with nerve and muscle diseases, at the 2012 annual meeting of the American Academy of Neurology in New Orleans, April 21-28.
The shorter-than-normal dystrophin protein produced via an experimental exon-skipping drug allows proper localization of proteins at the muscle-fiber membrane
Shortened versions of the muscle protein dystrophin — produced by skipping a section of genetic instructions called exon 51 — appear to be functional, says a new report from the United Kingdom.
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.
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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
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).