University of Miami Health System and Holtz Children’s Hospital physicians are playing a major role in getting ELEVIDYS access.
The therapy delivers a functional micro-gene to muscle cells, potentially arresting DMD progression and, in some cases, possibly reversing it.
Raniya was the first female ever treated with ELEVIDYS.
Siblings Raniya, who is 15 years old, and Amir, 8, received their single ELEVIDYS infusions at Holtz Children’s Hospital in late October. They may have to wait several months to learn if the treatment was successful, but early signs are encouraging.
“At her recent appointment, Raniya was already doing better, feeling stronger, walking more and not relying on a wheelchair as much,” said Dr. Lopez-Alberola.
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