Researchers look at the 'window of opportunity' for effective treatment in spinal muscular atrophy and the therapeutic potential of celecoxib

posted on June 20, 2013 - 4:27pm
Recent spinal muscular atrophy (SMA) research includes findings that shed additional light on the optimal "window of opportunity" for treatment and point toward a potential candidate for therapeutic development.

A new zebrafish research model may speed myofibrillar myopathy research; published results of a gene transfer study in LGMD are now accessible

posted on July 18, 2012 - 9:37am
Update (Aug. 8, 2012): This story was updated to reflect the availability of a podcast on the dysferlin gene transfer study. Zebrafish research models mimic myofibrillar myopathy

Are myelin and axons 'talking' to each other — and what are they saying?

posted on July 1, 2011 - 3:52pm
QUEST Vol. 18, No. 3
MDA-supported research in Charcot-Marie-Tooth disease is focused on figuring out what goes wrong at the molecular level in CMT-affected axons or the myelin sheaths that surround them, rather than on attempting to fix the problem directly or preserving nerve function in spite of it. A central theme emerging from the last decade of research is that myelin and axons require constant signals from...

A screen of more than 1,120 chemicals in dystrophin-deficient zebrafish revealed three candidates for further testing as treatments for Duchenne or Becker MD

posted on March 24, 2011 - 7:41am
Three small molecules that normalize muscle structure and function and improve survival in dystrophin-deficient zebrafish have been identified by scientists at Children's Hospital Boston, Harvard Medical School, and Alaska Fisheries Science Center in Juneau. The three chemicals, which already are approved for other medical uses, were found to increase survival and normalize muscle structure and...