CureDuchenne and Makes $1.5 Million Grant to Create DMD Clinic at CHOC

CureDuchenne has entered into a partnership with Children’s Hospital of Orange County to establish the CureDuchenne Clinic within the Neuromuscular Program at the hospital to provide state-of-the-art, multidisciplinary care and the latest advancements in diagnostics and treatment.

Duchenne is a severe, progressive, degenerative muscle disease. It is caused by mutations in the Duchenne gene, which encodes for dystrophin, a protein involved in muscle cell structure and signaling pathways. Without dystrophin, muscles throughout the body degenerate and become weak, eventually leading to loss of movement and independence, required support for breathing, cardiomyopathy and premature death.

The nonprofit CureDuchenne will provide CHOC nearly $1.5 million over three years to fund the CureDuchenne Clinic. CHOC, which is part of Rady Children’s Health, will build on decades of expertise to establish world-class care and research, integrate cutting-edge therapies, clinical trials, and expert-led services to enhance both quality of life and long-term outcomes for individuals with DMD and other neuromuscular diseases.

The partnership will expand CHOC’s multidisciplinary neuromuscular program to offer a range of specialties. It will work to advance the use of next-generation diagnostics for earlier and more precise disease monitoring, accelerate access to gene therapies and emerging treatments through its Outpatient Infusion Center, which will be located in CHOC’s new nine-story outpatient tower in Orange, California. It will also develop a national model for adult transition, ensuring long-term, comprehensive care as patients age.

In addition, the program will work to expand participation in clinical trials and research initiatives to ensure more individuals have access to promising therapies and scientific advancements. And it will create standards of care, including prioritizing the concerns of low-income families facing health inequities.

The CureDuchenne Clinic will be led by a dedicated team of leading specialists under the direction of Julian Thomas and John Crawford, Medical Director of neurology at CHOC and co-medical director of CHOC Neurosciences Institute.

“Comprehensive care from an experienced, multidisciplinary healthcare team is critical to addressing the complex needs of children living with Duchenne,” said Debra Miller, founder and CEO of CureDuchenne. “Through our partnership with CHOC, we are creating a seamless, patient-centered approach that integrates expert clinical care with groundbreaking research and innovative treatments.”

Photo: Debra Miller, founder and CEO of CureDuchenne