Duchenne caregivers have learned to focus on the person, not the disability

Suneel Ram’s needs gradually became greater after he was diagnosed at age 3 with Duchenne muscular dystrophy, a muscle-wasting genetic disorder that afflicts one in 5,000 boys.

Today, Ram, 28, requires physical and occupational therapies, help with personal hygiene and special food preparation, and depends on accessible spaces, a motorized wheelchair and specially equipped van.

Taking care of Ram has been a challenge, one that millions who have a loved one with physically debilitating illnesses also experience. But there are lessons and resources that can help make their tasks manageable and affordable, while making the quality of life more meaningful for caregivers and those in their care.

• Affected − but not defined − by muscular dystrophy: ‘We’re all the same, even if we don’t look like it’

In the case of Ram’s parents, Raghu Ram and Neera Gulati, family physicians who are divorced, the challenges and the uncertainty appeared overwhelming at first.

“When Suneel was young, I was always thinking about the future, and what was going to occur and when it was going to occur,” Raghu Ram said. “Sometimes, it just overwhelmed me. Try as best as you can to live in the moment, and not try to figure out or worry so much about what comes next.”

Their most valuable tip was to enroll in the Medicaid waiver program, Gulati said. The program, also known as a Home and Community-Based Services waiver, helps support people in their homes with disabilities and chronic health conditions.

Help from the program includes payment for aides, home assistance with ramps and wider doorways, vehicle modifications and no co-pays on most medicines.

Families diagnosed with Duchenne or other life-limiting disabilities qualifies for the Medicaid waiver program, regardless of income.

“You will need this waiver program no matter what,” Gulati said. “God bless New York State. This is actually something truly wonderful that they do.”

Organizations that help families get on the Medicaid waiver program include People Inc. and Aspire.

Ram’s condition was discovered through pediatrician visits at three-month intervals, allowing his development to be observed. Ram then began physical and occupational therapy in an early intervention program.

“See your pediatrician and family medicine doctor for all the routine preventative care visits,” Gulati said. “When the doctor sees there is a delay, they will know what to do.”

In October 2023, Gov. Kathy Hochul signed legislation making New York the second state to mandate Duchenne screening for newborns. An aide to Hochul credited a visit the then-lieutenant governor paid to a Suneel’s Light Foundation event years earlier as an influence in her decision to sign the legislation into law.

“That tells you there are effective medications now,” Gulati said. “The earlier the child is diagnosed, the earlier they can be treated.”

Photos: Navigating life with Duchenne muscular dystrophy

Here are other recommendations from Gulati:

• Treat people with Duchenne like everybody else. Don’t focus on the disability. Focus on what they can do.
• Carefully track doctor appointments, medications and equipment.
• Quality aides will change the quality of life for the person they are helping. Seek competent people who are considerate and compassionate.
• Get involved with the different Duchenne organizations, such as Parent Project Muscular Dystrophy, Cure Duchenne and Charley’s Fund.
• Every student should have an individual education plan (IEP), because it makes it possible to plug into school services, including the use of an aide.

“It’s important that the person with the disease, especially if they are an adult, is on board with the treatment plan,” Suneel’s father said.

“I think I take a little bit more of the position of trying to get Suneel to express what he wants, especially as he has gotten older,” Ram said.

Pavani Ram, Suneel’s stepmother, said medical advancements continue to offer hope.

“The pace that medical science is advancing is tremendous, and so we can’t know what’s coming necessarily,” Ram she said. “That’s another reason to live in the moment and really treasure what you have now.”

Participating in medical trials and going to the Duchenne Program at UMass Chan Medical School in Worcester can also offer significant benefits.

Ram and his mother, along with an aide, drove four times this year to the University of Wisconsin-Milwaukee to receive the trial drug Capricor he has taken for two years. The drug takes cardiac stem cells from cadavers and is used to slow Duchenne’s progression by assisting the heart and upper extremities.

They also go annually to the Duchenne Program run by Dr. Brenda Wong. Eleven foundations, including Suneel’s Light, came together to establish her clinic.

“There is more knowledge about the disease there than anywhere else in the world,” said Ken Manning, Suneel’s stepfather, though he and Gulati are divorced. “Dr. Wong knows more about this than anybody, period, and there isn’t even a close second.”

Wong, who has seen Ram since he was 11, discussed diet and how to avoid diabetes since he was in a hyperglycemic, pre-diabetic stage during a June visit. There were discussions on using a chest strap to stabilize his posture, and an abdominal binder to support his spine, and new trials Wong is aware of and whether any would be right for Ram.

Prolonged discussions were held with a specialist about how Ram can sleep more comfortably with the mask he wears at night, and an overnight sleep study was conducted. A respiratory therapist reported a slowing down of the disease’s progression, and a cardiologist was satisfied by tests on Ram’s heart.

Ram said it was reassuring to be there and get a comprehensive update on how he’s doing. He, his mom and stepfather believe the clinical trial is adding to Ram’s well-being, even if it’s not easily measurable.

Unlike their last visit, he was excited to get out of the medical center in time before the drive home to attend a show at the Ecotarium Museum of Science and Nature, also in Worcester.

When the lights came on in the Alden Planetarium after watching colorful images of Mars and the solar system crossing the screen, Ram’s eyes glowed.

“If people believe in me, I’m still going to keep going forward with some of my goals in life,” he said later, “with the faith that eventually it will work out.”