New York, Aug. 23, 2025 (GLOBE NEWSWIRE) -- The Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) today announced the publication of jointly developed consensus recommendations for the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy (DMD). This collaborative effort brings together the expertise of clinicians, researchers, and advocacy leaders to provide a standardized, practice-informed framework for patient selection, institutional readiness, safety monitoring, and follow-up. The full consensus guidelines, produced in partnership with cardiologists from the Advanced Cardiac Therapies Improving Outcomes Network (ACTION Network) and experts from 15 leading MDA and PPMD Clinical Care Centers, are published in Neuromuscular Disorders here.
Gene transfer therapy represents a promising advancement in the treatment of DMD but holds significant risk as well. With the FDA approval of the first gene therapy for the condition in 2023 and additional therapies in the pipeline, there is an urgent need for guidance to ensure that appropriate patients can access these treatments safely and equitably.
"These recommendations are the result of a truly collaborative effort between the medical and advocacy communities to protect patients while advancing access to innovative treatments," said Sharon Hesterlee, PhD, interim President and CEO, Muscular Dystrophy Association. "By providing a clear, consensus-based roadmap, these guidelines will help care centers navigate the complexities of gene therapy administration, from patient evaluation to post-infusion monitoring, giving families more information for their decision making"
The guidelines emphasize:
- 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.
"With gene therapy now available for Duchenne, it is critical that there be consistent, evidence-informed standards for how these therapies are administered and monitored," said Pat Furlong, Founding President, Parent Project Muscular Dystrophy. "These guidelines provide clinicians with a unified framework for evaluating eligible patients, delivery of the therapy, and support for patients during and following infusion. By working to establish clear protocols, we can help ensure safety, consistency, and confidence in the delivery of these complex therapies."
Dr. Barry Byrne, MD, PhD, Director of the Powell Gene Therapy Center at the University of Florida and member of the MDA Board of Directors, led the team to develop these guidelines stated, "Gene therapy offers hope for altering the course of Duchenne muscular dystrophy, but the safe and effective delivery requires specialized expertise and vigilant follow-up. These consensus guidelines are an important step toward unifying care practices, closing knowledge gaps, and supporting research that will refine these guidelines as our understanding evolves."
Families may contact the MDA Resource Center and the MDA Gene Therapy Support Network for guidance and support and learn more about the MDA Care Center Network. Families can also visit PPMD’s Gene Therapy Hub for insights and guidance to help you better understand the dynamic landscape of gene therapy. Learn more about PPMD’s Certified Duchenne Care Centers here.
Media Inquiries Contact:
MDA press@mdausa.org.
PPMD emily@parentprojectmd.org.
About Muscular Dystrophy Association
Muscular Dystrophy Association (MDA) is the #1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and over 300 other neuromuscular conditions. For 75 years, MDA has led the way in accelerating research, advancing care, and advocating support and inclusion of families living with neuromuscular disease. MDA's mission is to empower the people we serve to live longer, more independent lives. To learn more visit mda.org and follow MDA on Instagram, Facebook, X, Threads, Bluesky, TikTok, LinkedIn, and YouTube.
About Parent Project Muscular Dystrophy (PPMD)
Parent Project Muscular Dystrophy fights every single battle necessary to end Duchenne.
We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won eight FDA approvals.
Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, Instagram, and YouTube.
Attachment
Mary Fiance, Vice President, Strategic Communications Muscular Dystrophy Association press@mdausa.org
