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Smith Hosts Critical Meeting of Constituents, Top NIH Doctors on Duchenne Muscular Dystrophy

WASHINGTON, D.C.— There is “both an ongoing need and reason for optimism” said Rep. Chris Smith (R-NJ) after hosting a meeting with top doctors and researchers at the National Institutes of Health (NIH) to explore the agency’s work regarding Duchenne Muscular Dystrophy (DMD) and other muscular dystrophies.

JOH Invited to Washington to meet with Congressman and Top Doctors

Also participating in the meeting held in Smith’s office were NJ-04 constituents James Raffone and David Schultz, who have children with DMD and who are each working through their own foundations to help find a cure.

“The NIH experts at our meeting said they believe a breakthrough for DMD is in reach,” said Smith who is leading the charge for more federal funding for DMD research.  “However, we also learned that 84 percent of the qualified grant applications for DMD research are shelved because of a shortfall in funding. The sooner we expand federal support to competent, unfunded studies, the sooner we may identify better treatment therapies and, one day, a cure.”

DMD is a progressive muscle disorder affecting around 300,000 boys worldwide; around 400 to 600 boys in the U.S. are born with DMD each year. It causes a loss of both muscle function and independence, with a 100 percent fatality rate occurring normally in the late teens or early 20s. There is currently no cure for DMD.

“Even a modest boost in funding directed to DMD could enhance the quality of life for the boys and families who suffer with this disease every day,” Smith said.

“It’s an honor to work side by side with Congressman Smith. He’s a man of his word and fights with the passion and determination of a parent living with a child who has Duchenne. Together, we will find a cure,” said Jim Raffone.

“We are grateful for Congressman Smith’s dedication to increase medical research funding for muscular dystrophy. We are in a race against time and an increase in funding is crucial to bring potential therapies to children diagnosed with Duchenne muscular dystrophy,” said David Schultz.

David and Maria Schultz, and James and Karen Raffone, have sons with DMD. Both families were initially told by doctors that nothing could be done for their sons; the Schultzes then founded Ryan’s Quest, and the Raffones founded the non-profit foundation JAR of Hope, both to raise money for DMD research and bring widespread awareness of muscular dystrophies.

In the meeting at Smith’s office on Wednesday, JAR of Hope and Ryan’s Quest were able to hear first-hand from NIH experts of promising research that is being conducted for DMD, mostly by the six federal Wellstone Muscular Dystrophy Research Centers around the country, by researchers at private institutions that have received NIH grants, and by small biotech businesses.

NIH experts present at the meeting were Walter Koroshetz, MD, Director of the National Institute of Neurological Disorders and Stroke (NINDS); Glen Nuckolls, PhD, Program Director at the NINDS Division of Neuroscience; Tom Cheever, PhD, Program Director of the Muscle Disorders and Therapies Program at the National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS); Heather Rieff, PhD, Health Science Policy Analyst at the Office of Science Policy and Planning of NINDS; and Lauren Mullman, Legislative Analyst at NIH.

Doctor Walter Koroshetz emphasized the importance of raising awareness of the dire circumstances of families living with DMD.

In the September Labor-HHS appropriations bill which passed Congress and became law, NIH received a $2 billion increase in medical research funding. The Labor-HHS Committee Report included language Smith championed to push NIH to significantly expand their research on DMD.

“No one should be satisfied with a 1-in-5 funding ratio especially when experts say significant progress is close by,” Smith said. “It is critical that we focus more resources on researching and treating DMD, giving hope to families like the Raffones and the Schultzes who are fighting so hard for a cure.”

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