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Some Science We're Supporting
Relaxin for Treatment of Duchenne Muscular Dystrophy

Investigators: Jeff Chamberlain, PhD; Ron Berenson, MD; and Michael Regnier, PhD.

Relaxin is a natural hormone, which has a number of biological effects that may be of benefit in Duchenne Muscular Dystrophy (DMD), including its ability to regenerate muscle, reduce scarring and improve cardiac function.

HT-100 is a delayed-response form of halofuginone hydrobromide, a derivative of a natural compound found in a species of plant. The drug is being developed for Duchenne by Massachusetts-based Halo Therapeutics, LLC [...]

Boston Children Hospital
The Kunkel laboratory has started a preclinical mouse study of a PDE inhibitor provided by Pfizer Pharmaceuticals. We have previously shown that this compound markedly improves the phenotype of dystrophin null zebrafish and performs substantially better than parallel treatment with the PDE 5 inhibitor sildenafil. When we moved our studies into mdx5cv dystrophin deficient mice, the drug performed nearly as well as sildenafil even though it was not delivered at the optimum dose. We have since tested a number of delivery approaches, which will allow us to deliver the drug to obtain optimum serum concentrations. [...]

COLUMBUS, Ohio, Nov. 17, 2014 /PRNewswire/ -- The International Duchenne Alliance announced today that it will allocate $500,000 to Jerry Mendell, M.D. and Milo Biotechnology to support the first ever follistatin gene therapy trial for Duchenne muscular dystrophy. The trial will be conducted by Mendell at Nationwide Children's Hospital (Columbus, Ohio). Mendell has recently published positive data on follistatin in Becker Muscular Dystrophy. [...] read more

The Duchenne Alliance commits $500,000 to Jerry Mendell M.D. and Milo Biotechnology to fund first Duchenne Follistatin Gene Therapy Trial