LOPD: Elevating care through patient insights

Bridging patient experiences and clinical insights

Are you attending WorldSymposium™ in February 2025? Attend this accredited symposium, underpinned by data from a new patient survey, to hear how information from people with late-onset Pompe disease (LOPD) can trigger meaningful changes in their care.

Not sure you can be there in person? Register below and we’ll let you know when the symposium recording becomes available to watch on demand, here on Medthority.

Chair Professor Benedikt Schoser (Germany), along with Dr. Priya Sunil Kishnani (USA) and Dr. Tahseen Mozaffar (USA), will present the survey results and consider how key findings, including underrecognized symptoms, underreported needs, and the psychosocial impact of LOPD, could influence the way healthcare professionals support their patients with this condition.

Agenda

Sign up for symposium recording on demand

Faculty

Professor Benedikt Schoser

Benedikt Schoser is senior consultant neurologist at the Friedrich-Baur-Institute, Ludwig-Maximilians-Universität (LMU) Munich, Germany. He is a member of the executive board of the World Muscle Society and member of the European Academy of Neurology panel for neuromuscular disorders.

Professor Schoser has a long-standing interest in the pathogenesis and histopathology of muscular dystrophies, myotonic dystrophies, and metabolic myopathies. He has a special interest in translational gene therapy of myotonic dystrophies and glycogen storage disorders.

Disclosures: Amicus Therapeutics, argenx, Astellas Pharma, AVROBIO, Kedrion Biopharma, Maze Therapeutics, PepGen, Sanofi, and Taysha Gene Therapies.

Dr. Priya Sunil Kishnani

Priya Sunil Kishnani is Chief of the Division of Medical Genetics in the Department of Pediatrics, and Professor of Molecular Genetics and Microbiology at Duke University Medical Center, Durham, North Carolina, USA. Her research focuses on long-term complications of Pompe disease, as well as glycogen and lysosomal storage disorders.

Disclosures: Amicus Therapeutics, Asklepios BioPharmaceutical, Inc. (AskBio), Baebies, Bayer, Sanofi–Genzyme, and Maze Therapeutics.

Dr. Tahseen Mozaffar

Tahseen Mozaffar is a Professor of Neurology, and Pathology and Laboratory Medicine, and the Director of the Division of Neuromuscular Disorders at University of California, Irvine, USA. He is the Principal Investigator for UCI-NEXT and the Lead Investigator for INSPIRE-IBM. Dr. Mozaffar’s research interests include several neuromuscular disorders: myasthenia gravis, immune myopathies, rare myopathies, and ultra-rare myopathies.

Disclosures: Alexion, Amicus Therapeutics, AnnJi Pharmaceutical, argenx, Arvinas, Asklepios BioPharmaceutical, Inc. (AskBio), Audentes Therapeutics, AVROBIO, Cabaletta Bio, Cartesian Therapeutics, Grifols, Horizon Therapeutics (now Amgen), Immunovant, Maze Therapeutics, ML-Bio, Momenta (now Janssen), Muscular Dystrophy Association, Myositis Association, National Institutes for Health, Ra Pharmaceuticals (now UCB), Sanofi–Genzyme, Shionogi, Spark Therapeutics, UCB, Valerion, and Zogenix (now UCB).

Registration form

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