A cure is at the heart of Michael's Cause

After their son, Michael, was diagnosed with Duchenne muscular dystrophy (DMD) at age 4, Theresa and Rob Capolongo channeled grief into action. Determined to push toward better treatments—and ultimately a cure—they founded Michael’s Cause, a grassroots, all-volunteer nonprofit based in Staten Island, New York, where they live with Michael and his two siblings.

Since 2011, Michael’s Cause has directly funded more than $3 million in DMD research and support, including $250,000 directed to the Duchenne Program at UMass Chan Medical School. Michael became a patient soon after the program launched in 2018, led by founding director Brenda Wong, MD, a physician the family describes as “brilliant” and whom they followed from her previous appointment.  

The Capolongos are incredibly grateful for the Duchenne Program and the care, support, and hope it brings to Michael and the entire DMD community.

“Contributions to UMass Chan are so important because the program serves over 400 families,” said Rob. “Support from organizations like ours and other partners keeps it going. This is our way of helping and giving back.”

Following a comprehensive care model, the Duchenne Program relies on philanthropy to sustain its high level of multidisciplinary services and advance new approaches to treatment and care through innovation. Michael’s Cause is one partner in a coalition of more than a dozen patient-driven organizations funding the program.  

Stephen Chrzanowski, MD, PhD, assistant professor and the new director of the Duchenne Program, has taken the helm from Dr. Wong after her remarkable tenure and is continuing to build the program as a hub for both exceptional clinical care and growing research efforts.

Currently, twelve industry-sponsored clinical trials are underway, with several more launching soon. The team is also conducting investigator-led projects aimed at improving outcomes, including national and international collaborations studying non-invasive digital biomarkers, psychological health, the use of artificial intelligence in DMD management, cardiac transplant guidelines, weight management, and delayed puberty in individuals with DMD.

“Families like the Capolongos remind us why this work matters. Their partnership allows us to deliver comprehensive care today, while also investing in the research needed to improve the next generation of individuals living with Duchenne,” said Dr. Chrzanowski.

A devastating diagnosis inspires action

The lead up to Michael’s diagnosis in 2010 will strike a chord with many families affected by DMD—a fatal, rapidly progressive muscle-wasting disorder. As an active preschooler, he began to complain about pain in his calves with increasing frequency. A blood test ordered by his pediatrician revealed alarmingly high levels of creatine phosphokinase, a type of enzyme that muscle cells in the human body need to function, indicating considerable muscle damage.

“The pediatrician knew right away that Michael had DMD,” said Theresa. Genetic testing and a muscle biopsy confirmed the diagnosis.

Facing a devastating disease, Theresa and Rob acted quickly and decisively, believing that biomedical innovation holds the key to a better life for Michael and thousands of young people living with DMD. Their efforts started with cord blood banking after the birth of their third child that same year—a daughter whom they named “Victoria” as a nod to “victory” over the disease.

They established Michael’s Cause soon after in 2011, raising critical funds for DMD research and clinal services, as well as sponsorship of adaptive vehicles, equipment and other family supports.

“We started Michael’s Cause mainly to support research and finding a cure for DMD,” said Theresa. “That’s our end goal, no matter what.”

Community events throughout the year support fundraising, including the signature Step for a Cure 5K Run and Walk each September, which coincides with National Muscular Dystrophy Awareness Month.  

As a rare disease that affects about one in 3,500 to 5,000 newborns, fundraising for DMD isn’t always easy.

“Although DMD is much more recognized today, I still get some blank stares when I mention it,” said Rob. “But overall, the community has been very supportive, and our committee and family have done so much to advocate and try to make difference.”

“Michael is a big part of our fundraising efforts,” said Theresa. “He’s been an inspiration to others, whether or not he realizes it.”

Rob and Theresa express deep appreciation for everyone who has supported them on their journey with Michael, now 19 and a recent high school graduate. Michael continues to inspire with his resilience and his commitment to helping others with DMD, including participation in three clinical trials.  

“On behalf of our family, we’re humbled by the support that we’ve gotten as a charity,” said Rob. “We can only hope that science and medicine will get us closer to a cure. By supporting those efforts, including the Duchenne Program at UMass Chan, we’re doing our best to make an impact.”