Harvard Medical School

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...

Mice with a severe SMA-like disease that were injected with a synthetic "antisense" molecule developed bigger, more structurally sound muscles than untreated mice

posted on March 15, 2011 - 1:31pm
A team of research scientists has found that mice with a disease resembling a severe form of spinal muscular atrophy (SMA) that were treated with a gene-modifying molecule produced more of a needed protein throughout their spinal cords; developed bigger, stronger muscles; and survived longer than expected.