Background:As gene therapy may be a consideration for a broad portion of patients with DMD based on recent FDA approvals, and several ongoing investigational programs use this approach, it is essential to develop consensus-based best practice recommendations for the incorporation of gene therapy into regular DMD-related care. The first iteration of these guidelines was led by a consortium of interested, expert clinicians gathered by MDA and PPMD leadership. Over a dozen providers experienced in the administration of gene therapy shared protocols and best practice recommendations currently employed at their institutions.
Objectives: Create a set of best practices for gene therapy administration in patients with Duchenne muscular dystrophy.
Results: There was wide variability in practices and protocols across centers administering gene therapy, however, consensus was reached on a core set of best practices. Best practices for patient selection, institutional readiness, pre-dosing evaluation, day-of protocols, and short term monitoring were agreed upon across experts. All agreed that gene therapy should only be administered at centers experienced in the long-term care of patients with Duchenne and with experience administering gene therapy. Substantial institutional resources are needed to safely dose gene therapy. Finally, the first three months post-administration were the most vital time for close monitoring of patients.
Conclusions: These initial consensus-based recommendations can inform the efforts of centers administering gene therapy and/or the development of clinical trial protocols for investigational products. Additional research is needed regarding gene therapy in non-ambulatory Duchenne patients and patients with compromised respiratory and cardiac function, appropriate and science-based vector shedding protocols, and long-term safety and efficacy of various gene therapy products.