RYAN'S QUEST

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HAMILTON, NJ 08690

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SCIENCE WE ARE SUPPORTING

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

 

COLUMBUS, OH, 11/17/14 /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.

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.

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 approach-es, which will allow us to deliver the drug to obtain optimum serum concentrations.

Families of Boys with Duchenne Muscular Dystrophy Band Together to Fund Research

BALTIMORE, MD 04/12/17 — Three grass-roots foundations founded by the families of young boys diagnosed with Duchenne mus-cular dystrophy (DMD) have jointly awarded a $100,000 grant to Kennedy Krieger Institute's Center... READ MORE

DMD Therapeutics Inc. Receives Funding from DMD Foundations to Develop DMD-813 for Treatment of Duchenne Muscular Dystrophy

SEATTLE, WA 02/24/17 — DMD Therapeutics, Inc., a start-up biotech company, is developing proprietary therapeutics for Duchenne muscular dystrophy (DMD), an orphan disease associated with serious morbidity and early mortality that has a major unmet need.  READ MORE

2016: A Letter to My Son

2017: The Story Continues

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Michael's Cause, Ryan's Quest, Save Our Sons, Pietro's Fight, and Foundation LaForce Provide Research Grant to Create Potential New Duchenne Treatment

 

HAMILTON, NJ 03/24/16 — Ryan's Quest, a New Jersey based non-profit organization dedicated to finding treatments and a cure for Duchenne muscular dystrophy (DMD) is thrilled to announce the collaboration of five global Duchenne foundations...   READ MORE