ORLANDO, Florida—Dozens of patient advocacy groups, including many focused on Duchenne muscular dystrophy (DMD), devote themselves almost entirely to raising funds to cure a rare and devastating disease.
Team Joseph, a nonprofit based in the Detroit suburb of West Bloomfield, Michigan, is different. It raises money for scientific studies, but also looks for immediate gaps or needs not being met by other patient organizations.
“We believe in the power of research and the hope for tomorrow, but also in living for today,” founder Marissa Penrod told Rare Disease Advisor on the sidelines of CureDuchenne’s 2022 Futures conference here.
Named after Penrod’s son, Joseph, who has DMD, the charity has given out more than $1 million to nearly 1000 families in the 3 years since the establishment of the Duchenne Family Assistance Program—a collaboration between Team Joseph and another nonprofit, the Little Hercules Foundation, based in Dublin, Ohio.
Penrod said that Joseph, now 19, was diagnosed at age 5, at a time when steroids were not yet standard-of-care and few people had ever heard of gene therapy.
“We’ve been nearly 15 years in the Duchenne world, and opportunities have grown exponentially in the last 15 years,” she said. “Now, when somebody is diagnosed, there are lots of options in terms of clinical trials. There’s so much in the pipeline, and so many companies committed to the Duchenne space.”
Reaching Needy Patients and Families
For the first 10 years of its existence, Team Joseph funded DMD research and nothing else. It was only after Joseph at age 12 lost the ability to walk—and began to use a power wheelchair full-time—that Penrod began to consider how she could more directly help families affected by the disease.
“Yes, we want to get rid of Duchenne, but what do we do right now in the moment?” she said. “I started to think about other nuances of the journey. I have good health insurance for Joseph, and a supportive community. I’m able to provide for him what I think are the essentials, good doctors, the right equipment. But a lot of families aren’t as fortunate. That kept me up at night.”
Deriding what she views as a “pay-to-play” system in which poorer families are deprived of treatments and access to specialized doctors, Penrod said she was determined to help Duchenne families with limited means.
Specifically, Team Joseph helps pay for wheelchairs and other equipment not covered by insurance, as well as home modifications, travel assistance to get to clinical trials and doctor appointments, and accessible vehicles and modifications. The charity also helps parents of patients with Duchenne navigate Medicaid waivers, housing loans, and other government programs, as well as obtain health insurance coverage and appeal denials.
Penrod, 55, recalled when she asked a neurologist at a big-city hospital how low-income families without good insurance manage to care for their boys with Duchenne.
“‘Marissa, they don’t,’ she said to me so succinctly. She said, ‘I have families who don’t come to their checkups because they can’t afford train fare into the city,’” Penrod said. “That was a turning point for me.”
All Families Encouraged to Apply
Penrod, a single mom with a background in public relations and speechwriting, also has a 26-year-old daughter and a 23-year-old son; both are healthy.
“When Joseph was diagnosed, the way for me to channel my heartache and grief was to get busy,” she said. “About 5 years ago . . . I started to wonder how people navigate this journey if they don’t have resources. That’s what led me to start a Duchenne assistance program.”
Team Joseph, which operates on an annual budget of around $1 million, encourages anyone who needs help to apply.
“We can help with things like equipment. Some people need a scooter, or they need us to help pay for part of a wheelchair, or a ramp, or a down payment for a van,” Penrod said. “We don’t know everybody’s personal situation, nor do I believe it’s our job to dig into that. Taking care of a child with Duchenne—or, quite frankly, any rare disease—cannot be something only for an elite group of people.”
The organization receives money from individual donors as well as pharmaceutical firms such as Audentes, PTC Therapeutics, Wave Life Sciences, and Sarepta Therapeutics. It also works closely with other patient groups, including Parent Project Muscular Dystrophy and CureDuchenne.
In February 2021, Penrod addressed Congress remotely as part of a webinar titled “Economic Burden of Rare Diseases in America: A Public Health Crisis.” She also has a twice-monthly podcast, “Making Our Way,” on Apple and Spotify, and she speaks regularly at patient advocacy conferences.
“For everything that Duchenne takes away from us, there’s so much that remains. We don’t have to fix everything,” she said. “I used to pray for a miracle, then I had a revelation. The miracle is already here, and it’s Joseph.”