Multidisciplinary Group Publishes Consensus Guidance for Use of Gene Therapy in Duchenne Muscular Dystrophy

Jane B. Taylor, MD, MsCR, FAAP, ATSF, professor of pediatrics and clinical director of the Division of Pediatric Pulmonary Medicine at UPMC Children’s Hospital of Pittsburgh, was a coauthor of a published consensus document on the clinical delivery of gene therapy for Duchenne muscular dystrophy (DMD). The guidance was developed by a multidisciplinary group of clinicians and researchers assembled by the Muscular Dystrophy Association and Parent Project Muscular Dystrophy. The review was published in Neuromuscular Disorders in September 2025.

Dr. Taylor’s clinical emphasis is treating pulmonary complications in children and adolescents with neuromuscular conditions. She is a member of the multidisciplinary UPMC Children’s Muscular Dystrophy Program.

“Gene therapy has moved into clinical care for DMD, and clinicians need shared guidance to help navigate patient selection, monitoring, and early safety considerations as the use of these therapies grow,” Dr. Taylor says.

As gene-based therapies are used more frequently in clinical practice, there is a need for standardized, evidence- and practice-informed recommendations to support patient safety and clinician decision making.

The recommendations covered in the review article fall into four areas that are important for clinical teams and hospitals involved in gene therapy treatments for patients with DMD. These include:

• Criteria and considerations for identifying patients who may be appropriate candidates for gene therapy.
• Structural and operational factors that enable safe delivery of gene therapy for DMD patients.
• Monitoring patients for clinical safety, particularly during the first three months after treatment.
• Identifying and managing complications that may develop in the post-treatment period.

Although only one gene therapy product for DMD has received approval from the U.S. Food and Drug Administration so far, the recommendations developed by the group also can be applied to the use of future gene therapy products in development for this and other populations.

Clinical Learning Highlights

Clinical learnings for clinicians from the guidance include the emphasis on experienced multidisciplinary teams, the need for real-time safety surveillance, and the importance of transparent reporting of outcomes and adverse events. These elements are intended to support clinician decision making and patient safety as gene therapy becomes more widely used.

“Because many of the potential complications can affect multiple organ systems, early post-treatment care depends on close coordination across specialties, including pulmonary medicine, to ensure issues are identified and addressed promptly,” Dr. Taylor says.

The consensus document also acknowledges that substantial knowledge gaps associated with gene therapy for treating DMD remain to be filled in. These include long-term safety profiles, durability of treatment responses, optimal timing of dosing, and how combination therapies and genetic background may influence a patient’s response to gene therapy.

“The recommendations provide a starting point for clinicians as gene therapy becomes part of routine care for DMD, with the understanding that guidance will continue to evolve as the evidence-base and experience evolves, along with the new therapeutic options as they are developed and approved,” Dr. Taylor says.

Reference

Wolff JM, Capocci N, Atas E, et al. Consensus Recommendations and Considerations for the Delivery and Monitoring of Gene Therapy in Patients with Duchenne Muscular Dystrophy. Neuromuscul Disord. 2025; 54, 106207.