Medical Professional Publications

Researchers Awarded $4.7M to Explore Gene Therapy in Duchenne Muscular Dystrophy

(From the February 2017 issue of Research Now)

Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), announced on January 6, 2017 a $2.2 million grant to be awarded to Jerry Mendell, MD, and Louise Rodino-Klapac, PhD, both principal investigators in the Center for Gene Therapy at The Research Institute at Nationwide Children's Hospital. 

>> Read the full news release provided by Parent Project Muscular Dystrophy here

This grant will support Drs. Mendell and Rodino-Klapac and their team's work in exploring gene therapy as a potential treatment for Duchenne. Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a commercial stage developer of innovative RNA-targeted therapeutics, also announced in January that it has entered a research agreement with Nationwide Children’s Hospital on their microdystrophin gene therapy program.

The initial trial, expected to go into Phase 1/2a trial in late 2017, will be conducted at Nationwide Children’s. PPMD has committed the $2.2 million grant to the trial, with support from additional Duchenne foundations and families. Sarepta will also provide support to the trial through a separate research agreement with Nationwide Children’s, and will have an exclusive option to license the program. PPMD's grant provided incentive for Sarepta to help expand and accelerate this opportunity.

>> Read the full news release in our News Room

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