Passionate fundraising efforts help advance research and care
Thursday, February 13, 2025
Since 2018, the Duchenne Program at UMass Chan Medical School has offered a patient experience that is unique in health care: both medically comprehensive and seamlessly integrated, with care in multiple specialties offered in one visit. Described as “a gift” to patients and families, the globally renowned clinical and research site is devoted exclusively to extending and enhancing the lives of people with Duchenne muscular dystrophy (DMD).
A fatal, rapidly progressive muscle-wasting disorder, DMD primarily affects boys and occurs in one in 3,500 to 5,000 newborns. While a devastating diagnosis, the outlook for DMD—including quality of life and hope for a cure—has improved dramatically over the last two decades, thanks in large part to fierce advocacy and philanthropy from the patient community.
This powerful, patient-driven quest for change led directly to the development of UMass Chan’s Duchenne Program, which was conceptualized by and established with funding from Charley’s Fund, a nonprofit started by Tracy and Benjamin Seckler of Alford, Mass., in 2004 when their son Charley was diagnosed with DMD.
“When Charley was diagnosed, we soon discovered that nowhere in the Northeast offered the comprehensive, interdisciplinary care he needed,” said Tracy Seckler. “And when a clinical trial opportunity arose, we had to drag Charley on a plane to yet another city, weekly, to participate.”
Harnessing extensive knowledge of the DMD landscape, Charley’s Fund undertook a multiyear due diligence investigation to determine what it would take to build a world-class DMD clinic and research site in the region. Key were finding a host institution that would be a partner in innovation and a visionary to serve as the founding clinic director.
The Charley’s Fund team was impressed by UMass Chan’s work on amyotrophic lateral sclerosis (ALS) and its experience “focusing on one thing and doing it really, really well,” as well as being “extremely entrepreneurial in their vision.”
Brenda Wong, MD, a pediatric neurologist and internationally preeminent DMD expert, came on board as founding director after leading the Comprehensive Neuromuscular Center at Cincinnati Children’s Hospital Medical Center for 19 years.
“It had been a dream of mine to have a clinic and research program that is fully dedicated to this disease and research into treatments and a cure,” said Dr. Wong. “We are committed to providing seamless, full-spectrum care for everyone from infants to adults with Duchenne muscular dystrophy.”
A comprehensive, coordinated, patient and family-centric care model
While considered a neuromuscular disease, DMD affects almost all systems of the body, requiring care from many specialists, including neurologists, cardiologists, pulmonologists, gastroenterologists, nephrologists, endocrinologists and orthopedists. The Duchenne Program brings these specialists together under one coordinated care system.
“We want to play the role of quarterback,” said Dr. Wong. “By offering a medical home, our team of multidisciplinary specialists helps patients and families navigate the complexities of the health care system and connect the dots between issues in the heart, lungs, muscles, bones and endocrine system.
“As I tell the boys, when you come for your annual visit, it’s like you’re a car coming into my garage for a tune-up. We look at every aspect of care to ensure the car is running well. We want patients to leave here ready to excel in school and work and enjoy life.”
Patient families express great reassurance in knowing this truly holistic support model is delivering the best care possible for their children.
“I could not have survived parenting and losing a child with Duchenne muscular dystrophy without the Duchenne Program at UMass Chan,” said Beth Harvey of Dover, Massachusetts, whose son JB was a patient until he passed away from a sudden cardiac event at 15 years old in August 2023. Harvey started JB’s Keys to DMD, a founding supporter of the Duchenne Program, following JB’s diagnosis.
“The team’s commitment to treating the whole person and supporting their families is unmatched. Through crises from debilitating anxiety to broken bones, they are responsive and willing to communicate with other providers, schools and community-based care to get the patient the best quality of life. When JB passed away, my mind flooded with what I could have done differently. Hearing from Dr. Wong and other trusted members of the team just hours later put those thoughts at bay in a way that only JB’s most trustworthy and competent provider could do.”
While visits to the clinic are intensive due to the multidisciplinary approach, patients express gratitude for the high level of compassionate care.
“I feel very comfortable under the care of Dr. Wong and her team,” said Michael C., an 18-year-old patient. “She gives me a sense of hope.”
Patient families help close the funding gap
The Duchenne Program continues to rely on philanthropic support to maintain its care model. The coalition of more than a dozen patient-driven organizations funding the program mirrors the determination of families, everywhere, who are expending extraordinary effort to advance DMD research, clinical care and other services.
Tonya and Steve Dreher launched Hope for Gus in 2005 when their son, Gus, was diagnosed with DMD. Their flagship annual event, Everest to End Duchenne, brings a team of fundraisers on a symbolic trek through the Himalayan mountains in Nepal to Mount Everest Base Camp in honor of loved ones with the disorder—who cannot do the journey themselves—while simultaneously raising essential funds.
Gus has been a patient of Dr. Wong’s since her time in Cincinnati. After the experience of traveling far from their home in Peterborough, New Hampshire, for care, helping to launch a dedicated, accessible DMD clinic in the Northeast became a top priority.
“We were fortunate in that we could afford travel expenses for the initial visit, and then after that a clinical trial covered them,” said Tonya. “But I kept thinking, who can afford to travel like this all the time? For some families, the lack of a regional clinic could mean choosing between subpar care, major credit card debt or worse.”
The Plymouth-based Jett Foundation, launched by Christine and Stephen McSherry after their son, Jett, was diagnosed with DMD, offers a range of direct programming in addition to supporting research and clinical services such as the Duchenne Program at UMass Chan, through which they are proud to partner with other DMD advocacy organizations.
“The Duchenne Program is an incredible reflection of solidarity from the Duchenne community,” says Eric Snyder, Jett Foundation executive director.
A brighter future for DMD patients
Now serving more than 400 families, the Duchenne Program is currently boosting efforts to drive innovation in patient care and therapeutic treatments.
Stephen Chrzanowski, MD, PhD, joined UMass Chan in July 2024 as an assistant professor and co-director of the Duchenne Program, where he is focused on expanding clinical trial capacity and driving original research. His background as a physician-scientist, and deep commitment to pediatric neuromuscular disorders, made him a first-round draft pick for the new position.
“Growing up, I witnessed my best friend’s older brother who had DMD, and the family manage his illness, and the lack of treatment available for him,” said Dr. Chrzanowski. “UMass Chan’s unique capacity for bench to bedside research, and the integrated clinical care model, make the Duchenne Program an ideal incubator for real innovation. I feel fortunate to be a part of history as we redefine the trajectory of this terrible illness.”
As the Duchenne Program amplifies its mission to build a brighter future for DMD through clinical advancements, the need for funding to support and sustain these critical efforts grows, too. To make a financial contribution, please contact Heather Maykel, senior major gifts officer, at Heather.Maykel@umassmed.edu or 508-948-9218, or via the website.