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New muscular dystrophy treatment shows promise in early study led by Children's National
Date:9/17/2013

g/research/faculty/bios/cgmr/nagaraju_k.aspx" target="_blank">Kanneboyina Nagaraju, DVM, PhD, the lead author of the study, and principal investigator in the Center for Genetic Medicine at Children's National. "It is becoming increasingly clear that membrane integrity and repair are crucial factors not only in muscle, but also in cardiovascular, neurodegenerative, and airway disorders. In-creasing pre-clinical data suggest that VBP15 may show efficacy in these other disorders and in many other indications where steroids are used." Dr. Nagaraju noted.

Initial clinical trials of VBP15 are planned, pending Food and Drug Administration (FDA) review and approval, anticipated in 2014. ReveraGen Biopharma, Inc., the first Children's National private spin-off company, is working closely with Children's National investigators to translate the pre-clinical findings to DMD patients. ReveraGen Biopharma collaborated with NIH/TRND on VBP15, which was selected through a national com-petitive process, one of the few inaugural compounds selected through this novel pro-gram.

"The partnership with NIH and support from many others is illustrative of the importance of private-public partnerships that are essential in expediting the development of orphan disease therapeutics," remarked Edward Connor, MD, CEO, and CMO of ReveraGen Biopharma and Director of Innovation Development at Children's National. "We are optimistic that the new drug will translate well to patients, and are moving this forward as quickly as possible, while assuring safety."


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Contact: Emily Hartman
ehartman@childrensnational.org
202-476-4500
Children's National Medical Center
Source:Eurekalert

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