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Gene Therapy to Treat Duchenne Muscular Dystrophy Receives FDA Approval

The Food and Drug Administration authorized the first gene treatment for some patients with muscular dystrophy on June 22.

An uncommon and deadly hereditary disorder called Duchenne muscular dystrophy causes the body's muscles to weaken and deteriorate over time. Dystrophin, a protein that aids in maintaining the body's muscle cells' health, is absent due to a condition caused by a gene abnormality.

People with DMD may experience symptoms like difficulty walking and running, frequent falls, fatigue, learning difficulties, heart issues due to the impact on heart muscle functioning, and breathing difficulties due to the weakening of respiratory muscles involved in lung function.

Today's approval addresses an urgent unmet medical need and is an important advancement in the treatment of Duchenne muscular dystrophy, a devastating condition with limited treatment options, that leads to a progressive deterioration of an individual's health over time.

- Director of the FDA's Center for Biologics Evaluation and Research, Peter Marks

However, the government denied the proposal to make the medication available to all kids with Duchenne muscular dystrophy — the most prevalent kind of terminal muscle disease — who could still walk.

Instead, until more significant proof of the therapy's safety and efficacy was available, the government limited access to kids ages four through five. The choice received conflicting responses. Campaigners, some physicians and researchers, and parents of kids with genetic abnormalities applauded the restricted permission. However, several people were dissatisfied that the medicine wasn't instantly made more readily available.

While some claimed that even the restricted permission was premature, others complimented the agency's caution.

The medicine will be made accessible as soon as feasible, according to Sarepta Therapeutics of Cambridge, Massachusetts, the firm that developed it. The Elevidys medication, as it is known, will cost each patient $3.2 million.

However, this expedited approval procedure is debatable because some businesses must fulfill their commitments to demonstrate that their therapies are effective. Recently, a medicine that had been authorized in this manner to prevent preterm delivery was removed after it was discovered to be ineffective.

The program's initial authorized gene therapy is a treatment for muscular dystrophy offered by Sarepta. Muscles are destroyed by the illness, which almost primarily affects boys. Before they reach puberty, most men end up in wheelchairs, and their hearts and lungs eventually fail.

Most patients pass away in their 30s or 40s, but gene therapy transports a gene to patients' muscles by injecting billions of harmless viruses inside a genetically altered drug.

What are some side effects of Elevidys?

Vomiting, nausea, severe liver damage, fever, and thrombocytopenia were the most frequently reported adverse effects in Elevidys patients. The patient's liver function should be checked before starting Elevidys medication and once weekly for the first three months following treatment. Serious immune-mediated myositis may develop in patients taking Elevidys.

Additionally, after using Elevidys in clinical studies, myocarditis and increases in troponin-I have been noted. Troponin-I levels should be checked before giving Elevidys and once a week for the first month following treatment.


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