STAT+: After second Sarepta death, Duchenne muscular dystrophy community is racked by recrimination and worry

**Headline:** Second Duchenne Gene Therapy Death Prompts Criticism and Calls for Persistence

**Introduction:** In a troubling turn of events, another patient undergoing gene therapy for Duchenne’s disease has died, sparking intense debates within the Duchenne community. Advocates of gene therapy are questioning the actions of the FDA, while a significant portion of parents and doctors still push forward in supporting the therapy’s ongoing use.

**Emerging Concerns over FDA’s Involvement**
The news of the second fatal incident associated with Duchenne’s gene therapy has raised significant concerns about the U.S. Food and Drug Administration’s oversight of the treatment. Many parents and doctors hold the belief that the FDA has not been doing enough to ensure the safety and efficacy of the drug being developed by Sarepta Therapeutics. Critics argue that the incidence of the second death points to the necessity for more stringent clinical trials and data scrutiny before unproven therapies could be made available to patients diagnosed with Duchenne Muscular Dystrophy (DMD).

**Continued Support for Gene Therapy**
Despite the recent tragic occurrence, a sizable group of parents remain resolute in advocating for gene therapy as a promising treatment option for Duchenne. DMD is a fatal genetic disorder characterized by progressive muscle degeneration and weakness, and currently, there is no known cure for it. The potential benefits of gene therapy for treating Duchenne – including the ability to halt or slow the progression of the disease – have led many parents to continue endorsing for its use, even in the face of these severe adverse events.

**Balancing Hope and Risk**
The Duchenne community is now in a delicate position, balancing hope and risk related to gene therapy. The situation underlines the critical role that both the biomedical industry and regulatory bodies play in delicately balancing the pursuit of promising new treatments against the safeguarding of patient safety. The response from the Duchenne community also underscores the stark reality faced by those battling life-threatening diseases – the urgent need for effective treatments will often mean tolerating an elevated degree of risk. The tragic loss of the second Duchenne patient receiving gene therapy underscores a much larger public health debate – how to most responsibly navigate the often-murky waters of experimental therapeutic avenues, in a compassionate and scientifically sound manner.

Source: statnews.com