The Duchenne Program at UMass Chan Medical School offers integrative approach to care

The Duchenne Program at UMass Medical School is providing an integrative approach to treating young patients living with Duchenne muscular dystrophy, the most common and aggressive form of muscular dystrophy. Launched in 2018, the program provides comprehensive clinical care by a multidisciplinary, coordinated team of medical professionals, each with a specific expertise required to care for DMD patients: cardiologists, pulmonologists, endocrinologists and orthopedists. The clinic also integrates other specialties as needed, including gastroenterology, nephrology and ophthalmology; and provides physical therapy, nutrition, child life and orthotics support.

A program of cutting-edge clinical trials investigating a variety of new therapeutic approaches is offered, while original research is underway to develop new insights and innovations to power ongoing improvement in care and research for Duchenne patients.

A three-partner collaboration made the clinic possible. The partners include UMass Medical School, known for incubating innovative research and quality care; founding director Brenda Wong, MD, who is recognized worldwide for her practice in treating DMD patients; and Charley’s Fund, a patient-founded nonprofit that, in 15 years since inception, has directed nearly $45 million to speeding research and plays a vital role in driving critical efforts such as this new clinic.

“Over the past 20 years, we have learned so much about DMD, including how to improve best care practices and which experimental therapeutics might change the course of the disease,” said Dr. Wong, professor of pediatrics and director of the Duchenne Program at UMass Medical School. “The time is now for a clinic and research site that is fully focused on caring for patients with DMD and helping to develop new treatments. I made the decision to move to Worcester to open this clinic with Charley’s Fund and UMass Medical School because I knew that we could offer a unique, innovative care model that will result in better outcomes for patients and their families.”

The Duchenne Program was conceptualized and is funded by Charley’s Fund, a nonprofit organization started by Tracy and Benjamin Seckler of Alford, Mass., in 2004, when their son Charley was diagnosed with Duchenne muscular dystrophy. At the time, DMD patients and their families had no treatment options and faced a very grim forecast. Today, multiple new treatments are in development—from medicines that alleviate symptoms to gene replacement and gene editing approaches that strike at the root of the problem. Charley’s Fund has grown into an organization of international impact, with an innovative problem-solving approach and commitment to ensuring that nothing stands in the way of promising science. This fighting spirit finds its origin in Charley Seckler, for whom Charley’s Fund is named. Charley recently graduated from high school and will attend New York University in the fall, a remarkable achievement for a young man whose bleak prognosis did not allow for dreams of college or adulthood.

“As a mom, I’ve always felt a hole in the Northeast when it comes to comprehensive, integrated care for kids and young adults with DMD,” said Tracy Seckler, co-founder and CEO of Charley’s Fund. “Many families who could afford to travel would fly out of state to seek care and participate in clinical trials. And as the field of DMD research matures and clinical trials proliferate, lack of a robust clinic with deep clinical trial capabilities here in the Northeast threatened to slow down the historic advancements that are currently in the making. We refuse to allow that to happen, and so we decided to build a program that could become part of the solution.”

After two years of due diligence and planning—including a thorough search for a host site and clinic director—Charley’s Fund made a commitment of $6 million to fund the first five years of the Duchenne Program at UMass Medical School. Two local nonprofit partners are supporting Charley’s Fund in this effort: JB’s Keys to DMD and Hope for Gus. 

Learn more about this unique program in this Voices of UMassMed podcast with Brenda Wong, MD, founding director of the program, and Tracy Seckler, co-founder and CEO of Charley's Fund. Or visit www.umassmed.edu/duchenne/.