With so much emphasis these days on exon-skipping drugs, gene therapies and other scientific breakthroughs to treat Duchenne muscular dystrophy (DMD), it’s easy to lose sight of the physical, emotional and financial burdens of actually caring for a child with the disease.
Just ask the 38 caregivers featured in a new white paper issued by Team Joseph, a nonprofit organization based in Detroit, Michigan.
“We don’t do things normal families do. We don’t go to the beach. We haven’t been on a date in 10 years,” Jeremy, who has a daughter with DMD, wrote for the paper. “We don’t go to sporting events, we don’t go see shows. It’s just too hard.”
Rick, a father of a boy with DMD, wrote, “My son breaks down from time to time. He asks me why he has to die, and I don’t know what to tell him.”
Revealing pain — and possibilities
The 64-page report is titled “Engulfed in Caregiving: A Portrait of Love, Endurance, and Unrelenting Need.” Copies were handed out at the Muscular Dystrophy Association’s 2025 Clinical & Scientific Conference recently held in Dallas, Texas.
In a note to readers, the study’s authors — Alison Clabaugh, Ph.D., and Marissa Penrod — say the point is to spur thought, to nudge collaboration, and to question the status quo.
“We hope you’re uncomfortable at times, filled with awe and hope, and fueled by outrage,” they write in the prologue. “We hope this is disruptive. Let it keep you up at night — maybe because you feel the pain and the problems, but mostly because you see the possibilities.”
Raw and unfiltered, the report features direct quotes from conversations with caregivers of boys and young men with DMD. In fact, most of the participants surveyed by Team Joseph were not identified at all; a minority were identified only by first name.
One father of a boy with DMD said, “It took us at least 6 of the last 8 years to get to where my wife is not crying all the time. That’s a tragedy for parents to be thrown into something like this. If you’re not strong enough and you don’t have enough support, it will destroy you.”
Penrod, the founder and CEO of Team Joseph — and the mother of Joseph, 22, who has DMD — said many parents inhabit what she called the “dark corners of Duchenne” and are simply not being heard.
“So we hired a research company, went out into the community, and conducted focus-group listening sessions to really document the lived experience of Duchenne with those who are underrepresented and really underserved,” she said.
In addition to being interviewed or participating in a focus group, 34 of the 38 caregivers included in the report completed a demographic survey. Of the total, 22 were biological mothers of the loved one with Duchenne, seven were biological fathers, three were adoptive parents and two were grandparents. The caregivers’ ages ranged from 35 to 82, with a median age of 45, while the median age of the loved one with Duchenne was 14.5 years.
While 26% of respondents reported annual household income of $75,000 or more, 36% said their annual family income was under $50,000. Likewise, 17 families — half of the total surveyed — said they lose wages when traveling to or attending medical care appointments with their child.
“Doctors sometimes won’t write a [prescription] without seeing him, so it’s an extra trip. Then I lose income for that day,” Rick said. “It costs me money to take him to those appointments, but what other choice do I have besides moving closer [to the center] or changing jobs?”
Research for the white paper — a project of the Duchenne Community Voices Initiative 2024 — was conducted by ThinkGen, a healthcare marketing firm, while support came from Catalyst Pharmaceuticals, Edgewise Therapeutics, ITF Therapeutics, NS Pharma, Pfizer, PTC Therapeutics and Sarepta Therapeutics.
The initiative intentionally seeks out Duchenne families from a range of cultural, geographic and socioeconomic backgrounds to share their stories, and enables that by hosting listening sessions in various cities so caregivers don’t have to pay for flights or lodging, Penrod said.
“This report is driven by photos of families and also direct quotes from them. When a caregiver says, ‘Insurance wouldn’t cover a patient lift for us, so I bought some scaffolding and built my own,’ that should keep us up at night,” Penrod told Rare Disease Advisor — sister brand to DMD Companion — adding that “money might not help you cure the disease, but it can help with quality of life.”
Exploring inequities
One common denominator is that — due to the progressive nature of the disease — most boys with Duchenne will eventually need a power wheelchair to get around. Yet being able to afford this expensive device, she said, “depends on your insurance coverage, and your capacity to spend time on the phone navigating an insurance battle.”
Learn more about DMD treatment and care
Particularly vulnerable, Penrod said, are families lacking transportation, and those living in rural areas far from treatment centers, as well as non-traditional caregivers such as single parents, grandparents and those with health issues of their own.
“I’m 5 hours away from Dallas where we have doctor’s appointments,” an unidentified parent said in the report. “It would be nice if somebody would actually put themselves in a family’s shoes where we’re experiencing this stuff.”
Such families often feel that the medical establishment ignores the wishes of patients themselves.
“Some doctors decide on the medications for my sons,” said Jennifer, who has six children, including three with Duchenne. “I want them to tell me the options and we’ll help our sons decide. Ask them what they want. People need to hear their voice.”
Black, Hispanic, and lower-income Americans — along with people whose first language is not English— are often left out of the conversation, according to the report.
Penrod admits that as a middle-aged white woman, “I see a lot of me at conferences like this one” — and suggests that the Duchenne community is not nearly as inclusive as it should be.
“People need to feel like they belong. There’s a place for them,” she said. “When we talk about representation, we need diversity in speakers on the stage. We need diversity in those who do advocacy. We need diversity in our nonprofits. We have to lead by example that we are actually inclusive, not just giving it lip service.”
Penrod said she hopes her organization’s report will be shared among lawmakers on Capitol Hill as well as insurance and pharmaceutical executives.
“The experience of those living with Duchenne is relevant to anybody who has a role in our lives or cares about the quality of life for our loved ones,” she said. “It’s payers. It’s regulators. It’s pharma companies. It’s advocacy groups. The idea is not that it stays with Team Joseph, but that we get it out into the world.”
For a free printed copy of the report, send a request to info@teamjoseph.org.
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