Parent Project Muscular Dystrophy Provides $250,000 in Funding to Rare Disease Research, LLC to Establish New Clinical Trial Site

WASHINGTON, Nov. 7, 2023 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), today announced that PPMD has provided $250,000 in funding to Rare Disease Research, LLC (RDR), 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 RDR's creation of an avenue that increases patient access to clinical trials while expediting the advancement of treatments for Duchenne.

RDR has strategically appointed Edward Smith, MD, a distinguished 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. Dr. Smith's exceptional 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.

Eric Camino, PhD, PPMD's Vice President of Research and Clinical Innovation, emphasizes the organization's innovative approach in advancing Duchenne treatments. "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. 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," he said. "We are excited for this opportunity to support Dr. Smith and RDR's efforts to expand their footprint. Doing so will increase access to clinical trials for patients and speed the process of finding treatments that will end Duchenne for every single person impacted by the disease."

Han Phan, MD, Head of Research and Principal Investigator at RDR, sheds light on the remarkable strides made by RDR and how this collaboration will help alleviate a travel burden many families experience. "RDR is conducting over 35 active clinical trials in rare disease indications at RDR in Atlanta, including several studies for Duchenne patients, and we see that more than 70% of our study participants fly to Atlanta from around the U.S. and even other countries, in order to participate in our 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, in clinical trials that can last for a couple of years. Therefore, we are thrilled to collaborate with PPMD to open an additional site in North Carolina, and improve access to clinical trials for the rare disease patients and reduce their burden of participating in clinical research," Dr. Phan states. "Living with rare conditions is uniquely challenging for patients, and we should make it as easy as possible to participate in clinical research and accelerate the development of new groundbreaking therapies faster," she added.

An open house event will be held at the new RDR site in Hillsborough, NC, on November 29, 2023, from 2:30-6:30 PM ET.

ABOUT PARENT PROJECT MUSCULAR DYSTROPHY:

Duchenne is a genetic disorder that slowly robs people of their muscle strength. Parent Project Muscular Dystrophy (PPMD) fights every single battle necessary to end Duchenne.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won seven FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. To learn more about PPMD's robust Research Strategy, click here. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube. 

ABOUT RARE DISEASE RESEARCH, LLC:

RDR is an independent clinical research center, 100% 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's team of expert physicians, researchers, and clinical staff works directly with rare disease foundations, parent networks, and leading academic researchers specializing in rare conditions. RDR's clinical research sites offer technology-enabled clinical research solutions tailored for rare disease clinical trials, including an accelerated study start-up process, overnight facilities for intensive phase I/II studies, IP pharmacy and research home health services for decentralized trials. To learn more about RDR, please visit rarediseaseresearch.com.

SOURCE Parent Project Muscular Dystrophy (PPMD)