Sarepta Therapeutics of Cambridge, Mass., has received word from the U.S. Food and Drug Administration (FDA) that the agency will consider an accelerated approval pathway for eteplirsen, an experimental drug the company is developing for boys and young men with Duchenne muscular dystrophy (DMD) who can potentially be treated by skipping of exon 51 of the dystrophin gene.
CHICAGO, April 14, 2014 – The Muscular Dystrophy Association, the largest nonprofit funder of neuromuscular disease research in the United States, has awarded funding for 38 new research grants totaling $3.6 million for the first year and $10.7 million over three years.The awards will be made to researchers seeking promising treatments and cures for muscle diseases within MDA's program.
NEWTON, Mass., and TUCSON, Ariz., Nov. 14, 2013 — Halo Therapeutics LLC and the Muscular Dystrophy Association (MDA) today announced that $500,000 has been awarded for development of the experimental drug HT-100 as a treatment for Duchenne muscular dystrophy (DMD).
MDA supported development of utrophin-boosting compounds for years
TUCSON, Ariz. — The Muscular Dystrophy Association announced today that SMT C1100, an experimental drug for Duchenne muscular dystrophy (DMD), has been found safe in healthy human volunteers and reached blood levels potentially adequate for a therapeutic benefit. Next steps are to move into trials in people with DMD.