Tannika Scott, Raniya's mother, shares the emotional challenges of caring for children with DMD and expresses her hope for the success of the new therapy.

The FDA has approved a new gene therapy called ELEVIDYS, specifically designed to treat older patients with Duchenne muscular dystrophy (DMD).

Duchenne muscular dystrophy is caused by mutations in the dystrophin gene, which leads to progressive muscle weakness and limited mobility.

ELEVIDYS employs a harmless virus to deliver a functional micro-gene to muscle cells, potentially halting or even reversing the progression of DMD.

Clinical trials indicate that ELEVIDYS can improve motor function, particularly in younger children, although older patients may encounter more significant challenges due to pre-existing muscle damage.

Looking ahead, Dr. Lopez-Alberola aims for advancements in DMD treatment that would allow for interventions before symptoms manifest, thereby improving overall patient outcomes.

Dr. Roberto Lopez-Alberola, a physician at the University of Miami, is optimistic about the therapy's effects on Raniya, who is 15 years old.

Scott, a parent of two children affected by DMD, recounts the struggles of obtaining a correct diagnosis for his daughter Raniya, who was initially misdiagnosed with diabetes.

Raniya and her younger brother Amir, aged 15 and 8 respectively, received their treatments in late October 2024, with early signs of improvement noted in Raniya's condition.

Patients undergoing treatment with ELEVIDYS must be closely monitored for side effects and require immunosuppression to prevent their immune systems from attacking the therapy.

Dr. Lopez-Alberola expresses hope that future FDA approvals will facilitate earlier treatment in infants, potentially preventing irreversible muscle loss.

Originally approved for children ages 4 and 5 in 2023, the FDA expanded the eligibility for ELEVIDYS to include older patients in June 2024.