PPMD, in collaboration with the Muscular Dystrophy Association (MDA), is proud to share the release of newly published consensus guidelines for the delivery and monitoring of gene therapy in Duchenne muscular dystrophy (Duchenne).
Developed in partnership with cardiologists from the Advanced Cardiac Therapies Improving Outcomes Network (ACTION Network) and experts from 15 leading PPMD Certified Duchenne Care Centers and MDA Care Centers, these guidelines provide a standardized, practice-informed framework for patient selection, institutional readiness, safety monitoring, and follow-up.
This work represents the combined expertise of the medical and advocacy communities, recognizing the importance of working to establish clear protocols that can help ensure safety, consistency, and confidence in the delivery of these complex therapies. The full consensus guidelines are now available in Neuromuscular Disorders.
Why These Guidelines Matter
While gene transfer therapy represents a promising advancement in the treatment of Duchenne, it also carries risks. With the FDA approval of the first gene therapy for Duchenne in 2023 and more therapies in the development pipeline, the Duchenne community urgently needs clear, evidence-informed standards to guide how these therapies are delivered.
For families and clinicians alike, having a roadmap provides clarity in a complex and evolving space. The goal of the guidelines is to protect patients, empower families with more information, and ensure that access to gene therapy is delivered in a way that is both safe and equitable.
What the Guidelines Emphasize
The consensus-driven recommendations outline best practices to help care centers navigate the complexities of gene therapy administration—from evaluating eligible patients through post-infusion monitoring. They highlight:
- The importance of experienced, multidisciplinary care teams.
- Standardized protocols for monitoring and managing adverse events.
- Real-time safety surveillance and transparent reporting.
- Strategies to address health equity, including language access, financial assistance, and geographic considerations.
Support for Families
Families seeking more information can check out:
- PPMD:
- MDA:
The post Newly Published Consensus Guidelines Aim to Ensure Safe and Equitable Delivery of Gene Therapy in Duchenne appeared first on Parent Project Muscular Dystrophy.