FDA Approves Gene Therapy for Rare Genetic Condition in Boys

The FDA has approved a gene therapy treatment for boys aged four and five with Duchenne muscular dystrophy, a rare and fatal genetic disorder that causes muscle degeneration. The treatment involves delivering a replacement gene through a disabled virus, and it has shown promising results in clinical trials. However, concerns have been raised about the high cost of the treatment, which is priced at $3 million. Despite this, families affected by Duchenne muscular dystrophy see the approval as a reason for hope and believe it will change the trajectory of the disease.