MDA and PPMD Release Consensus Guidelines for Safe and Equitable Use of Gene Therapy in Duchenne

Advertisement News|Articles|September 13, 2025 MDA and PPMD Release Consensus Guidelines for Safe and Equitable Use of Gene Therapy in Duchenne Author(s)Marco Meglio New guidelines enhance gene therapy delivery for Duchenne muscular dystrophy, emphasizing multidisciplinary care and safety monitoring for optimal patient outcomes. Advertisement Through a joint effort of clinicians, researchers, and advocacy leaders, the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) announced the publication of new guidelines that promote the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy (DMD).1,2DMD is caused by mutations in the DMD gene, one of the largest in the human genome, which encodes dystrophin, a critical protein for stabilizing muscle fibers. To date, the FDA has approved one gene therapy, Sarepta’s Elevidys, an AAV vector-based therapy that is indicated for both ambulatory and non-ambulatory patients with DMD.While Elevidys remains the only approved gene therapy, the published guidelines are potentially applicable to other products in clinical development. The main themes of the guidelines include the value and role of multidisciplinary care teams, protocols for monitoring and managing adverse events, and safety surveillance and transparent reporting."One of the most important take-home messages is that gene therapy requires an amplified version of the multidisciplinary care we already deliver in Duchenne," Barry Byrne, MD, PhD, chief medical officer of the MDA, told NeurologyLive®. "Beyond the routine involvement of neurology and cardiology, we now need hematology, nephrology, immunology, and other subspecialists engaged from the very beginning."Byrne, who also serves as director of the Powell Gene Therapy Center at the University of Florida, added, "That’s because the adverse events we’ve observed are often immune-mediated, and only a team fully aligned on protocols and communication can both prevent and respond to these complications in real time."The consensus guidelines emphasize institutional readiness, which includes having access to rapid labs and weekly or same-day tests, as well as consistency across different administration sites. Per the new guidelines, institutions should have an established plan for rare complications, begin to build rapport with payers, and proactive prepare for authorization requests with documented eligibility.In terms of patient selection, eligible patients for gene therapy should have a confirmed genetic diagnosis with updated molecular testing, as well as a review of exclusions according to the therapy’s label. Patients must be on high dose corticosteroids for at least 2 months before starting treatment and must undergo weekly blood tests and routine laboratory tests for safety."We now recommend close weekly monitoring of liver function tests—AST, ALT, and GGT—following gene therapy, since subacute inflammation and endothelial injury have been seen despite routine glucocorticoid use," Byrne noted.During the preparing stages for gene therapy, the recommended guidelines note several baseline labs, such as troponin-1, CMP, CBC, and liver panel, as well as cardiac and evaluations and functional assessments, such as the North Star Ambulatory Assessment, PuL, and 10-Meter Walk/Run. At this stage, patients should be informed about consent obligations, as well as a rundown of their future care plan and what it will involve.On the day of treatment, clinicians should be prepared to review the patient’s medical history, prep infusion sites, and be vigil of and infusion reactions. Post-infusion observation is expected to last around 3 hours, and institutions should be equipped with discharge instructions and an emergency plan if necessary.In the early stages following the start of gene therapy, the guidelines call for weekly labs testing troponin, platelets, and LFTs, followed by a corticosteroid taper that starts around 8 weeks. Safety remains the most key part during this 3-month phase, as clinicians should track adverse events, including cardiac toxicity, TMA, and hepatoxicity, while also adjusting ECG monitoring based on symptoms. For adjustments, the group of experts recommended following Action Network recommendations, while also noting that troponin elevation 3 times more than baseline warrants immediate follow-up. In addition, arrhythmia should trigger a cardiac consultation.Past the initial 3-month stage, the guidelines call for continued tracking of gene therapy safety and efficacy through annual lab panels, as well as ensured AE reporting through the FDA Adverse Event Reporting System (FAERS) and registries like ENDURE."The number one takeaway of all this is that we have to ensure that these products are safe in order to fully realize the direct benefit they might bring to patients. That’s why establishing best practices around safety became such a central theme of the guidelines," Byrne added. "There’s growing interest in agents like rapamycin, already used in transplantation, which may enhance the safety of therapy. The hope is not only to reduce the rate of liver toxicity but also potentially increase efficacy, which could fundamentally change the risk-benefit conversation for patients and families."READ MORE: Claseprubart Heads to Phase 3 Study of Myasthenia Gravis Following Positive MaGic TrialLiver toxicity, as Byrne noted, was the subject of recent concern for Elevidys and other Sarepta gene therapy products. In mid-July, the FDA requested the company to suspend the distribution of Elevidys following the third death of a patient in Sarepta’s gene therapy program. The latest death, a 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3, was enrolled in the company’s phase 1 study testing SRP-9004, also known as patidistrogene bexoparvovec. This third instance was also attributed to liver failure, the same as the prior 2 deaths.3At the time, the FDA revoked Sarepta’s platform technology designation and met with the company to request a voluntary halt on all Elevidys shipments. Sarepta declined to comply with the agency’s request. The reasons for revoking were because of insufficient evidence—especially in light of new safety concerns—that the company's AAVrh74 Platform Technology can be used across multiple drugs without compromising safety.Less than 2 weeks after the FDA shut down the distribution of Elevidys, the agency recommended to remove the voluntary hold for ambulatory patients eligible for the gene therapy. At the time, the FDA’s investigation concluded that the death of the ambulatory 8-year-old boy, who was on Elevidys, was unrelated to the gene therapy product itself. The agency also noted it will work with Sarepta regarding non-ambulatory patients, which remains subject to a voluntary hold, following 2 deaths.4Regarding the future landscape of gene therapy, Byrne went on to note, that “We now know that more than a thousand boys with Duchenne have already received gene therapy, and additional products are right behind. Some of the most exciting work involves gene editing approaches that could apply to at least half the Duchenne population, or near full-length dystrophin strategies that are mutation independent."He added, "Others aim to improve tissue targeting—enhancing muscle or cardiac delivery while lessening liver toxicity. Together, these advances suggest that patients will soon have multiple options, hopefully available at younger ages, and supported by the best safety practices outlined in these recommendations."REFERENCES1. Muscular Dystrophy Association and Parent Project Muscular Dystrophy Announce Joint Consensus Guidelines for Safe and Equitable Delivery of Gene Therapy in Duchenne Muscular Dystrophy. News release. Muscular Dystrophy Association. https://www.mda.org/press-releases/mda-and-ppmd-announce-joint-consensus-guidelines2. 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. Neuromuscular Disord. Published online August 23, 2025. doi:10.1016/j.nmd.2025.1062083. FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths. News release. FDA. July 18, 2025. Accessed September 12, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold4. FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients. News release. FDA. July 28, 2025. Accessed September 12, 2025. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients NewsletterKeep your finger on the pulse of neurology—subscribe to NeurologyLive for expert interviews, new data, and breakthrough treatment updates.Subscribe Now! Advertisement Related ArticlesDORAs Carry Lower Real-World Abuse, New Phase 3 Argus Data, RAP-219 Meets Primary End PointSeptember 13th 2025NeurologyLive® Friday 5 — September 12, 2025September 12th 2025Shared Decision-Making and Ethics in Complex Neurology Clinical Research: Paul Ford, PhDSeptember 12th 2025FDA Hands Complete Response Letter to SL1009 for Pyruvate Dehydrogenase Complex DeficiencySeptember 12th 2025Expanding the Alzheimer Drug Development Pipeline September 12th 2025Special Episode: Understanding Fremanezumab's Expanded Indication in Pediatric MigraineSeptember 12th 2025 Latest CMEMultimediaBurst CME™ Part II: The Evolving Treatment Landscape for Huntington DiseaseSamuel A. 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Advertisement Through a joint effort of clinicians, researchers, and advocacy leaders, the Muscular Dystrophy Association (MDA) and Parent Project Muscular Dystrophy (PPMD) announced the publication of new guidelines that promote the delivery and monitoring of gene therapy in patients with Duchenne muscular dystrophy (DMD).1,2DMD is caused by mutations in the DMD gene, one of the largest in the human genome, which encodes dystrophin, a critical protein for stabilizing muscle fibers. To date, the FDA has approved one gene therapy, Sarepta’s Elevidys, an AAV vector-based therapy that is indicated for both ambulatory and non-ambulatory patients with DMD.While Elevidys remains the only approved gene therapy, the published guidelines are potentially applicable to other products in clinical development. The main themes of the guidelines include the value and role of multidisciplinary care teams, protocols for monitoring and managing adverse events, and safety surveillance and transparent reporting."One of the most important take-home messages is that gene therapy requires an amplified version of the multidisciplinary care we already deliver in Duchenne," Barry Byrne, MD, PhD, chief medical officer of the MDA, told NeurologyLive®. "Beyond the routine involvement of neurology and cardiology, we now need hematology, nephrology, immunology, and other subspecialists engaged from the very beginning."Byrne, who also serves as director of the Powell Gene Therapy Center at the University of Florida, added, "That’s because the adverse events we’ve observed are often immune-mediated, and only a team fully aligned on protocols and communication can both prevent and respond to these complications in real time."The consensus guidelines emphasize institutional readiness, which includes having access to rapid labs and weekly or same-day tests, as well as consistency across different administration sites. Per the new guidelines, institutions should have an established plan for rare complications, begin to build rapport with payers, and proactive prepare for authorization requests with documented eligibility.In terms of patient selection, eligible patients for gene therapy should have a confirmed genetic diagnosis with updated molecular testing, as well as a review of exclusions according to the therapy’s label. Patients must be on high dose corticosteroids for at least 2 months before starting treatment and must undergo weekly blood tests and routine laboratory tests for safety."We now recommend close weekly monitoring of liver function tests—AST, ALT, and GGT—following gene therapy, since subacute inflammation and endothelial injury have been seen despite routine glucocorticoid use," Byrne noted.During the preparing stages for gene therapy, the recommended guidelines note several baseline labs, such as troponin-1, CMP, CBC, and liver panel, as well as cardiac and evaluations and functional assessments, such as the North Star Ambulatory Assessment, PuL, and 10-Meter Walk/Run. At this stage, patients should be informed about consent obligations, as well as a rundown of their future care plan and what it will involve.On the day of treatment, clinicians should be prepared to review the patient’s medical history, prep infusion sites, and be vigil of and infusion reactions. Post-infusion observation is expected to last around 3 hours, and institutions should be equipped with discharge instructions and an emergency plan if necessary.In the early stages following the start of gene therapy, the guidelines call for weekly labs testing troponin, platelets, and LFTs, followed by a corticosteroid taper that starts around 8 weeks. Safety remains the most key part during this 3-month phase, as clinicians should track adverse events, including cardiac toxicity, TMA, and hepatoxicity, while also adjusting ECG monitoring based on symptoms. For adjustments, the group of experts recommended following Action Network recommendations, while also noting that troponin elevation 3 times more than baseline warrants immediate follow-up. In addition, arrhythmia should trigger a cardiac consultation.Past the initial 3-month stage, the guidelines call for continued tracking of gene therapy safety and efficacy through annual lab panels, as well as ensured AE reporting through the FDA Adverse Event Reporting System (FAERS) and registries like ENDURE."The number one takeaway of all this is that we have to ensure that these products are safe in order to fully realize the direct benefit they might bring to patients. That’s why establishing best practices around safety became such a central theme of the guidelines," Byrne added. "There’s growing interest in agents like rapamycin, already used in transplantation, which may enhance the safety of therapy. The hope is not only to reduce the rate of liver toxicity but also potentially increase efficacy, which could fundamentally change the risk-benefit conversation for patients and families."READ MORE: Claseprubart Heads to Phase 3 Study of Myasthenia Gravis Following Positive MaGic TrialLiver toxicity, as Byrne noted, was the subject of recent concern for Elevidys and other Sarepta gene therapy products. In mid-July, the FDA requested the company to suspend the distribution of Elevidys following the third death of a patient in Sarepta’s gene therapy program. The latest death, a 51-year-old man with limb-girdle muscular dystrophy (LGMD) type 2D/R3, was enrolled in the company’s phase 1 study testing SRP-9004, also known as patidistrogene bexoparvovec. This third instance was also attributed to liver failure, the same as the prior 2 deaths.3At the time, the FDA revoked Sarepta’s platform technology designation and met with the company to request a voluntary halt on all Elevidys shipments. Sarepta declined to comply with the agency’s request. The reasons for revoking were because of insufficient evidence—especially in light of new safety concerns—that the company's AAVrh74 Platform Technology can be used across multiple drugs without compromising safety.Less than 2 weeks after the FDA shut down the distribution of Elevidys, the agency recommended to remove the voluntary hold for ambulatory patients eligible for the gene therapy. At the time, the FDA’s investigation concluded that the death of the ambulatory 8-year-old boy, who was on Elevidys, was unrelated to the gene therapy product itself. The agency also noted it will work with Sarepta regarding non-ambulatory patients, which remains subject to a voluntary hold, following 2 deaths.4Regarding the future landscape of gene therapy, Byrne went on to note, that “We now know that more than a thousand boys with Duchenne have already received gene therapy, and additional products are right behind. Some of the most exciting work involves gene editing approaches that could apply to at least half the Duchenne population, or near full-length dystrophin strategies that are mutation independent."He added, "Others aim to improve tissue targeting—enhancing muscle or cardiac delivery while lessening liver toxicity. Together, these advances suggest that patients will soon have multiple options, hopefully available at younger ages, and supported by the best safety practices outlined in these recommendations."REFERENCES1. Muscular Dystrophy Association and Parent Project Muscular Dystrophy Announce Joint Consensus Guidelines for Safe and Equitable Delivery of Gene Therapy in Duchenne Muscular Dystrophy. News release. Muscular Dystrophy Association. https://www.mda.org/press-releases/mda-and-ppmd-announce-joint-consensus-guidelines2. 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. Neuromuscular Disord. Published online August 23, 2025. doi:10.1016/j.nmd.2025.1062083. FDA Requests Sarepta Therapeutics Suspend Distribution of Elevidys and Places Clinical Trials on Hold for Multiple Gene Therapy Products Following 3 Deaths. News release. FDA. July 18, 2025. Accessed September 12, 2025. https://www.fda.gov/news-events/press-announcements/fda-requests-sarepta-therapeutics-suspend-distribution-elevidys-and-places-clinical-trials-hold4. FDA Recommends Removal of Voluntary Hold for Elevidys for Ambulatory Patients. News release. FDA. July 28, 2025. Accessed September 12, 2025. https://www.fda.gov/news-events/press-announcements/fda-recommends-removal-voluntary-hold-elevidys-ambulatory-patients NewsletterKeep your finger on the pulse of neurology—subscribe to NeurologyLive for expert interviews, new data, and breakthrough treatment updates.Subscribe Now! Advertisement Related ArticlesDORAs Carry Lower Real-World Abuse, New Phase 3 Argus Data, RAP-219 Meets Primary End PointSeptember 13th 2025NeurologyLive® Friday 5 — September 12, 2025September 12th 2025Shared Decision-Making and Ethics in Complex Neurology Clinical Research: Paul Ford, PhDSeptember 12th 2025FDA Hands Complete Response Letter to SL1009 for Pyruvate Dehydrogenase Complex DeficiencySeptember 12th 2025Expanding the Alzheimer Drug Development Pipeline September 12th 2025Special Episode: Understanding Fremanezumab's Expanded Indication in Pediatric MigraineSeptember 12th 2025 Latest CMEMultimediaBurst CME™ Part II: The Evolving Treatment Landscape for Huntington DiseaseSamuel A. 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Musiek, MD, PhD; Malú Gámez Tansey, PhD View moreIn-Person + Virtual EventPutting the Patient First in Acute Pain Management: The PA’s Guide to Incorporating Cutting-Edge Science into their Treatment StrategiesOctober 9, 2025View moreIn-Person Event4th Women in Neurology ConferenceOctober 24-26, 2025Register now!VideoMedical Crossfire® - Optimizing Management for Patients With Generalized Myasthenia Gravis: Focus on Complement InhibitorsJames F. Howard Jr, MD; Nicholas J. Silvestri, MD, FAAN; Neelam Goyal, MDView moreVideoMedical Crossfire®: Integrating Real-World Data to Improve Outcomes for Patients With Multiple SclerosisCarrie M. Hersh, DO, MSc, FAAN; Anna Shah, MDView moreMultimediaCommunity Practice Connections™: Optimizing the Management of Tardive Dyskinesia—Addressing the Complexity of Care With Targeted TreatmentIlan Melnick, MD; Alejandro Alva, MD; Linda Trinh, DNP, PMHNP, FNP, MPHView moreMultimediaBurst CME™: Optimizing Migraine Management – Addressing Unmet Needs, Individualizing Care for Diverse Populations, and Utilizing CGRP Targeted AgentsJessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHSView moreMultimediaBurst CME™: Setting the Stage – Individualizing Migraine Care for Diverse Populations Across Care SettingsJessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHSView moreMultimediaBurst CME™: The Patient Journey – Unmet Needs From Diagnosis Through Management of MigraineJessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHSView moreMultimedia Evolving Perspectives in Alzheimer Disease : Reaching an Earlier Diagnosis, Understanding Neuroinflammation, and Exploring Therapeutic AdvancesAlireza Atri, MD, PhD; Erik S. Musiek, MD, PhD; Malú Gámez Tansey, PhDView moreMultimediaBurst CME™: Optimizing the Use of CGRP Targeted Agents for the Treatment of MigraineJessica Ailani, MD, FAHS, FAAN; Paul G. Mathew, MD, DNBPAS, FAAN, FAHSView moreVirtual EventExpert Illustrations & Commentaries™: Visualizing the Role of B Cells as Therapeutic Targets for Generalized Myasthenia GravisOctober 31, 2025View moreVideoCases and Conversations™: Applying Best Practices to Prevent Shingles in Your PracticePaul G. Auwaerter, MD, MBA; Paul P. Doghramji, MD, FAAFP; Aruna Subramanian, MDView moreVideoPatient, Provider, and Caregiver Connection™: Pediatric Myasthenia Gravis—Current Treatment and Emerging ConceptsJohn F. Brandsema, MD; Abigail N. Schwaede, MD; Samuel J. Mackenzie, MD, PhDView moreAudioMind Moments™: Optimizing Management for Patients with Generalized Myasthenia GravisJames F. Howard Jr, MDView moreVideoCases and Conversations™: Navigating the Complexities of Managing Myasthenia Gravis in Pediatric and Pregnant Patient PopulationsJohn Brandsema, MD; Neelam Goyal, MDView moreVideoIdentifying and Treating CIDP in the Modern EraJeffrey A. Allen, MD; Karissa L. Gable, MDView moreVideoCases and Conversations™: Little Voices, Big Challenges – Comprehensive Care for Pediatric Spinal Muscular AtrophyJohn Brandsema, MD; Carla D. Zingariello, DOView moreVideoA Breath of Strength: Managing Cancer Associated LEMS and Lung Cancer as OneEstelamari Rodriguez, MD, MPH; Millie Das, MD; Ruham Nasany, MDView moreVideoStriking the Right Nerve: Managing Cancer Associated LEMS in Lung Cancer PatientsAnne Chiang, MD, PhD; J. Douglas Miles, MD, PhD, FAAN; Suma Satti, MDView moreVideoPatient, Provider, and Caregiver Connection: Improving Diagnosis and Care for Adolescent Myasthenia Gravis Patients – A New FrontierAbigail N. Schwaede, MD; Emmanuelle (Noelle) Tiongson, MDView moreVideoA New Era in NMOSD Treatment: Optimizing Therapeutic Transitions and Reducing Patient BurdenShamik Bhattacharyya, MD, MS, FAAN; Mirla Avila, MD; Dean Wingerchuk, MDView moreAudioPER Podcast™: Maximizing the Multidisciplinary Care Outcomes of Patients with Amyotrophic Lateral Sclerosis (ALS)Richard Bedlack, MD, PhDView moreAudioPER Podcast™: Best Strategies for Approaching the Diagnosis of Amyotrophic Lateral Sclerosis (ALS)Terry Heiman-Patterson, MDView moreVideoPER Postgame™: Updates from ANNA in the Management of IgA NephropathyGerald Bernard Appel, MD; Ellie Kelepouris, MD, FACP, FAHAView moreVideoPatient, Provider, and Caregiver Connection: Turning a New Leaf in Acute Pain Management – How Recent Advancements Impact the Treatment ParadigmJeffrey Gudin, MD; Jeffrey Bettinger, PharmDView morePrevious slideNext slide Advertisement Advertisement Trending on NeurologyLive1MDA and PPMD Release Consensus Guidelines for Safe and Equitable Use of Gene Therapy in Duchenne2Expanding the Alzheimer Drug Development Pipeline 3DORAs Carry Lower Real-World Abuse, New Phase 3 Argus Data, RAP-219 Meets Primary End Point4NeurologyLive® Friday 5 — September 12, 20255FDA Hands Complete Response Letter to SL1009 for Pyruvate Dehydrogenase Complex Deficiency