Mom Fights FDA to Save Son with Rare Disease

Angelina's decades-long battle to find a treatment for her family's battle with Duchenne muscular dystrophy

Apr. 12, 2026 at 1:30pm

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Angelina, a mother from El Paso, Texas, has been fighting Duchenne muscular dystrophy for over 40 years. She lost two of her brothers, Angelo and Antonio, to the terminal illness, and her 14-year-old son, Ryu, is now also battling the disease. Angelina recently visited Washington, D.C. to share her family's story with lawmakers and patient advocates, urging the FDA to approve a gene therapy treatment called Elevidys that could help save her son's life. Her advocacy efforts have already led to the resignation of a key FDA official who had blocked access to such treatments.

Why it matters

Duchenne muscular dystrophy is a rare and fatal genetic disorder that primarily affects young boys. Families like Angelina's have long struggled to access potentially life-saving treatments due to regulatory hurdles and bureaucratic roadblocks at the FDA. Angelina's story sheds light on the human cost of these delays and the urgent need for the FDA to prioritize approving safe and effective therapies for rare diseases.

The details

Angelina has been caring for family members with Duchenne muscular dystrophy since childhood. Her brothers Angelo and Antonio both died from the disease in their 20s. When Angelina's son Ryu was diagnosed as a toddler, she added him to her daily prayers for healing. This year, Angelina was given the opportunity to share her family's story with lawmakers and patient advocates in Washington, D.C., urging them to push the FDA to approve the gene therapy treatment Elevidys, which could potentially save Ryu's life. Angelina's advocacy efforts have already led to the resignation of Dr. Vinay Prasad, a key FDA official who had blocked access to such treatments.

  • Angelina's brothers Angelo and Antonio died from Duchenne muscular dystrophy in their 20s.
  • Ryu, Angelina's son, was diagnosed with the same disease as a toddler.
  • This year, Angelina visited Washington, D.C. to share her family's story and advocate for FDA approval of the Elevidys gene therapy treatment.
  • Dr. Vinay Prasad, who had blocked access to treatments like Elevidys, announced his resignation from the FDA just a week after Angelina's advocacy efforts.

The players

Angelina

A mother from El Paso, Texas who has been fighting Duchenne muscular dystrophy for over 40 years, losing two of her brothers to the disease and now advocating to save her 14-year-old son Ryu.

Angelo

Angelina's brother who died from Duchenne muscular dystrophy at age 20.

Antonio

Angelina's brother who died from Duchenne muscular dystrophy at age 22.

Ryu

Angelina's 14-year-old son who is currently battling Duchenne muscular dystrophy.

Dr. Vinay Prasad

A former FDA official who had blocked access to treatments like Elevidys and recently announced his resignation from the agency.

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What they’re saying

“This year, that prayer was answered when I was asked to speak out not just on behalf of my brothers and son, but for every family that feels isolated because of a terminal rare disease.”

— Angelina

“I'm just a mom. But we recently celebrated Easter, where a carpenter saved the world. He overcame the establishment of His time, which was willing to throw the vulnerable and sick to the side. He fell, but He didn't falter — I hope to follow His example.”

— Angelina

“As we were approaching Holy Thursday this year, Ryu was having a hard evening. He needed his Bipap machine to help his lungs function, as he so often does. But he looked at me — my 14-year-old wheelchair-bound boy who is the happiest kid I know — and said, 'Mom, this sucks. But what you're doing makes it a lot easier.'”

— Angelina

What’s next

Angelina plans to continue advocating for FDA approval of the Elevidys gene therapy treatment that could potentially save her son Ryu's life. She is hopeful that the resignation of Dr. Vinay Prasad, who had blocked access to such treatments, will pave the way for greater progress.

The takeaway

Angelina's decades-long fight to find a treatment for her family's battle with Duchenne muscular dystrophy highlights the urgent need for the FDA to prioritize approving safe and effective therapies for rare diseases. Her advocacy efforts, which have already led to the resignation of a key FDA official, demonstrate the power of a single voice to create change and save lives.