PPMD Provides $250,000 in Funding to Rare Disease Research to Establish New Clinical Trial Site

Rare Daily Staff    

Parent Project Muscular Dystrophy, a nonprofit organization leading the fight to end Duchenne muscular dystrophy, has provided $250,000 in funding to Rare Disease Research to open a second independent rare disease clinical research site in the Raleigh-Durham metropolitan area in North Carolina.

With this funding commitment, PPMD aims to support Rare Disease Research’s (RDR’s) creation of an avenue that increases patient access to clinical trials while expediting the advancement of treatments for Duchenne.

RDR is an independent clinical research center, 100 percent dedicated to conducting clinical research, accelerating the development of safe and effective treatments for rare diseases, and providing access to innovative investigational therapies to patients with rare diseases.

RDR is conducting over 35 active clinical trials in rare disease indications at RDR in Atlanta, including several studies for Duchenne patients. More than 70 percent of our study participants fly to Atlanta from around the United States and other countries to participate in its clinical trials.

“This is a significant burden on the patients and their families, especially in studies that may require weekly or monthly treatment or assessments, and in clinical trials that can last for a couple of years,” said Han Phan, head of research and principal investigator at RDR.

RDR has appointed Edward Smith, a pediatric neurologist renowned for his expertise in neuromuscular clinical trials, as the principal investigator and clinical director of the new trial site in Hillsborough, NC. Smith’s background includes an impressive tenure as a faculty member in the Duke Departments of Pediatrics and Neurology from 2007 to 2023. During this period, he founded and directed the Duke Children’s Neuromuscular Program, exemplifying his commitment to providing comprehensive, multidisciplinary care to patients with muscle and nerve disorders.

“PPMD takes a comprehensive view of the drug development landscape to identify opportunities to accelerate the development of therapies and reach as many patients as possible,” said Eric Camino, PPMD’s vice president of research and clinical innovation. “This includes supporting a variety of approaches, including novel trial design and management to make participation in clinical research as accessible for as many individuals as possible.”

Photo: Eric Camino, PPMD’s vice president of research and clinical innovation