Enhancing Gene Therapy Safety for Duchenne Through New Recommendations: Barry Byrne, MD, PhD

WATCH TIME: 3 minutes

"The No. 1 takeaway is that we must ensure safety. Only by closely monitoring and managing immune-related events can we fully realize the benefits gene therapy offers patients with Duchenne."

Elevidys (Sarepta), also known by its investigational name delandistrogene moxeparvovec-rokl, made history as the first gene therapy approved for Duchenne muscular dystrophy (DMD), receiving accelerated FDA approval in June 2023. This innovative therapy, which gained full traditional approval less than a year later, uses an adeno-associated virus (AAV) vector to deliver micro-dystrophin gene, a shortened yet functional version of the dystrophin protein, into muscle cells, thus working to restore the dystrophin essential for muscle integrity.

Recently, through a collaboration between the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy, a group of experts published new consensus guidelines for safe and equitable use of gene therapy in DMD. The recommendations, which apply to Elevidys and other products in clinical development, reiterate the importance of experienced, multidisciplinary care teams, standardized protocols for monitoring and managing adverse events, and real-time safety surveillance and transparent reporting. In addition, the group of clinicians, researchers, and advocacy leaders touch on strategies to address health equity, including language access, financial assistance, and geographic considerations.

Following the publication of these new guidelines, NeurologyLive® sat down with senior author Barry Byrne, MD, PhD, to discuss some of the top-line considerations clinicians should take away and how to apply them to clinical practice. Byrne, a chief medical adviser to the MDA, outlined the standard protocols for monitoring adverse events related to gene therapies, highlighting the importance of weekly liver function testing and the potential role of adjunctive agents like rapamycin to mitigate risks and improve therapeutic efficacy.

Click here to view the 2025 consensus recommendations and considerations published in Neuromuscular Disorders.