First Female in the World Receives Revolutionary New Gene Therapy for Muscular Dystrophy in Miami

When Raniya Scott was just two years old, her world changed with an unexpected diagnosis: Duchenne muscular dystrophy (DMD), a group of diseases that causes muscles to weaken and break down over time. The news was overwhelming for her young mother, and it puzzled her doctors, as this rare and progressive genetic disorder primarily affects boys, not girls.

Raniya’s DMD affected everything from walking and breathing to blood circulation. For 11 years, Raniya has depended on medication, physical therapy, and a wheelchair to help manage her worsening symptoms.

But now, a new gene therapy is offering the family hope. Raniya, now 16, is the first female in the world to receive the groundbreaking gene therapy, thanks to physicians at UHealth – University of Miami Health System and Holtz Children’s Hospital. Additionally, her younger brother, Amir Monstanto, 8 – who also has DMD – received the treatment alongside her.

What is Elevidys and how the gene therapy works
Elevidys, the first and only FDA-approved gene therapy to treat DMD, introduces a functional micro-gene into muscle cells through a harmless virus, potentially halting the progression of DMD and, in some cases, even reversing it.

The treatment is given through a simple infusion, but patients must be immunosuppressed to prevent their immune response from destroying the therapeutic virus, which can lead to complications.

So far, early signs are promising.

In clinical trials, the drug has demonstrated an ability to improve motor function in younger children. Since DMD is a progressive disease, older patients typically have more muscle damage, which can make it more challenging for the treatment to be effective.

“The worst-case scenario is that some patients do not regain any function. However, we may slow down the disease’s progression. That alone would be transformational,” said Roberto Lopez-Alberola, M.D., chief of pediatric neurology at UHealth, and a neurologist at Holtz Children’s Hospital. “At Raniya’s recent appointment, she was already doing better, feeling stronger, walking more, and not relying on a wheelchair as much.”

The FDA initially approved the therapy for children ages 4 and 5, but in June 2024, it expanded to include older children. While it may take months to determine if the treatment is successful, doctors say early signs are encouraging.

“I have been working in neurogenetics for 30 years, and it’s amazing to see the dream of gene therapy being realized,” Dr. Lopez-Alberola said. “It’s a wonderful opportunity to treat these patients and see the impact it’s having. There is much to celebrate.”

Life with DMD
Raniya and Amir live in Naples and frequently travel with their mom, Tannika Scott, to Miami for appointments with Dr. Lopez-Alberola. Caring for two children with progressive disease presents ongoing challenges.

For Raniya, one of the most difficult challenges came when she transitioned to using a wheelchair—a moment that marked a significant shift in her journey.

“It’s very hard, especially seeing them falling,” Scott said. “She had to get into a wheelchair, and she kept asking why she has to do this.”

The family has a history of DMD, with two of Scott’s uncles suffering from the condition. But unlike her uncles, she is hopeful the new research and treatments will lead to a cure for her children.

“At first, I was scared and nervous,” Scott said of the new treatment. However, after research and trust in her team at Holtz Children’s and UHealth, she knew she had to give it a try. “I’m hoping Raniya and Amir can try to live a normal life.”

Hoping for a brighter future
A few weeks after the treatment, Scott is hopeful for the research that could ultimately change her children’s lives. She’s grateful to the teams at Holtz Children’s and UHealth for their unwavering support and continued care.

“I just thank them, and I thank God that I met them, and they love my kids and want the best for my kids,” she said. “They are a very good team. I wouldn’t trade them for anything. Dr. Lopez-Alberola is the best doctor who really cares about his patients, and this is something that he wanted for a long time.”