AMDA Continuing Medical Education –
CME Partnerships

Late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly. More can present subtly and progress slowly, making early diagnosis a clinical challenge. Misdiagnosis can lead to delayed treatment initiation and increased morbidity and mortality. While newer therapies such as enzyme replacement therapy (ERT)A treatment that replaces the missing enzyme through IV infusion. More have expanded the treatment landscape and improved the natural course of LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More and patient quality of life, early initiation of treatment when clinical or imaging signs first appear is key to optimizing outcomes.

Join us for this program, in which a panel of neurology experts and a nurse practitioner will review emerging therapeutic options and share practical approaches to timely diagnosis and treatment initiation. Through a multidisciplinary lens, the discussion will explore team-based strategies and shared decision-making that support patient-centered care. A compelling pre-recorded patient vignette will highlight the lived experience of LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More, helping to connect clinical decision-making with real-world impact.

• Identify patients with late-onset Pompe disease (LOPD)A form of Pompe disease that begins after infancy and usually progresses more slowly. More using the latest diagnostic criteria and tools, such as GAA enzyme-activity testing and genetic analysis
• Review the differences in the mechanisms of action and long-term impacts on motor and respiratory function of available therapies for LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More
• Summarize the practical implications of the latests safety and efficacy findings of newly approved therapies for LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More, including recent trial results and patient-reported outcomesInformation about health, symptoms, or quality of life reported directly by patients. More
• Discuss individualized treatment plans that incorporate multidisciplinary careCare provided by a team of health professionals from different specialties working together. More collaboration and shared decision-making for patients with LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More

• When to suspect LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More
• Clinical clues, biomarkersA measurable sign in the body that helps track disease progression or treatment response. More, and genetic testingA test that looks for changes in a person's genes, including changes in the GAA gene. More
• Epidemiology, genetic underpinnings, burden of disease
• From mechanism to patient outcomes: Navigating the expanding landscape of LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More therapies
• Overview of current treatment options
• Emerging therapeutic options
• Where newer therapies fit in the treatment algorithm
• Personalizing team-based care and treatment through shared decision-making
• Coordinating care within the multidisciplinary teamCare provided by a team of health professionals from different specialties working together. More
• Practical considerations of integrating newer therapies into LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More care

Release Date: September 11, 2025- enduring only

Expiration Date: September 11, 2026

The educational design of this activity addresses the needs of neurologists, neuro-muscular HCPs (MDs, DOs, NPs, PAs, nurses), pediatric neurologists, pediatricians, orthopedists, physical therapists, respiratory specialists, and other HCPs involved in the care of Pompe diseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More.

Tahseen Mozaffar, MD, FAAN
Professor of Neurology
University of California, Irvine

Tahseen Mozaffar, MD, FAAN is a professor of neurology and pathology and laboratory medicine and the director of the Division of Neuromuscular Disorders at the University of California, Irvine (UCI). He is the associate director for the Center for Translational Sciences Award (CTSA) at UCI, and is the Principal Investigator for UCI-NEXT, a NeuroNEXT award funded by NINDS/NIH. He is actively involved in clinical and translational research in neuromuscular disorders, including currently serving as Principal Site Investigator on over a dozen clinical trialsA research study that tests new treatments or approaches in people. More in myasthenia gravis, rare and ultra-rare myopathies, and in immune myopathies. He has co-authored over 190 peer-reviewed publications and has authored or co-authored over a dozen book chapters and invited reviews.

Benedikt Schoser, MD
Professor of Neurology
Friedrich-Baur-Institute Dep. of Neurology
LMU Clinic Munich Germany

Benedikt Schoser, MD is a neurologist, neurophysiologist, neurointensivist, palliative medicine doctor, and muscle pathologist. He is a professor of neurology and a senior consultant neurologist at the Friedrich-Baur-Insititute’s Department of Neurology at the Ludwig-Maximilians-University in Munich, Germany. His research interests are in multisystemic neuromuscular disorders, translational research, and therapy. He is the executive section editor of Neuromuscular Disorders and a member of the editorial board of Current Opinions in Neurology and the European Journal of Neurology. He has authored more than 380 peer-reviewed publications in clinical and translational science.

Natalie Goedeker, DNP, CPNP
Co-director, pediatric neuromuscular clinical research
Washington University in St. Louis School of Medicine

Natalie Goedeker, DNP, CPNP is a pediatric nurse practitioner in the department of neurology, neuromuscular division, at Washington University School of Medicine in St. Louis. She is the co-director of the Pediatric Neuromuscular Clinical TrialsA research study that tests new treatments or approaches in people. More Team. She has over 10 years of experience caring for pediatric patients, most of which has been spent caring for children with neurological and neuromuscular diseases. She has investigator experience in over 30 clinical trialsA research study that tests new treatments or approaches in people. More for pediatric neuromuscular diseases and helps coordinate care for patients receiving gene transfer therapy, enzymeA protein that helps the body carry out chemical reactions. More replacement therapies, and other complex therapeutics in the neuromuscular space.

Dwayne M. Wilson
Pompe Patient

Dwayne M. Wilson was diagnosed with Late Onset Pompe DiseaseA form of Pompe disease that begins after infancy and usually progresses more slowly. More (LOPDA form of Pompe disease that begins after infancy and usually progresses more slowly. More) in 2018 after years of unexplained symptoms. He lives in Irvine, California, with his wife and mother-in-law and is a devoted father and grandfather. Since starting treatment, Dwayne has embraced advocacy, becoming a patient speaker, MDA Ambassador, and former columnist for Pompe DiseaseA rare genetic disease in which the body cannot properly break down glycogen, leading to buildup tha... More News. He shares his story to raise awareness and spread hope—living by the motto: “Pompe, it’s in my DNA. I have Pompe, Pompe doesn’t have me.”