The patient died after using $ 3 million gene therapy

The gene therapy is called Elevidys, provided by Sarepta Therapeutics. In the official announcement, Sarepta said the young patient died from acute liver damage, a side effect that was previously reported. However, this is the first time the company has recorded such a serious illness. Therapy has been used on more than 800 patients.

Sarepta said the patient has recently been infected. This may be the reason he damages the liver. The company said they had plans to update prescribed information for Elevidys to reflect this case.

Elevidys uses an inactive virus to bring the gene to replace Dystrophin production into the patient cell. However, the cost of treatment is quite expensive, once the transmission costs 3.2 million USD. In addition, Elevidys also potentially side effects such as hepatitis, liver enzyme and immune response with viral vector. Sarepta is continuing to test to expand the indication of an older group of patients, opening up hope for a step further in the treatment of genetic disease with gene technology.

In 2023, despite concerns about efficiency and safety, the US Food and Drug Administration (FDA) approved the use of Elevidys to treat Duchenne muscle dysplasia. This is the first gene therapy approved in the US for this rare disease causing this muscle atrophy. The disease causes men to weaken muscle, lose their ability to exercise and die early.

Last year, the FDA licensed Elevidys completely, expanding the target group as a patient from 4 years old, regardless of whether they are still able to travel or not. In the past, therapy was only for younger patients, and the ability to exercise.

Since 2016, the FDA has quickly approved three drugs to treat Duchenne muscle disorders of Sarepta Company. No drug has been confirmed as effective. Experts are still conducting research to ensure FDA conditions.