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Trachea Cells under the Microscope

Accredited symposium: Elevating LOPD care

Supported by Amicus Therapeutics
Last updated: 24th Feb 2025
Published:24th Feb 2025

Watch our expert-led accredited symposium focused on translating patient voice into improved outcomes in LOPD and earn 1 CME credit.

How much do you know about overlooked symptoms and unmet needs in LOPD? Take this short quiz to find out and access the full symposium. We’ll ask you again at the end to see what you recall.

1. Pompe disease extends beyond a muscular disorder, affecting multiple facets of an individual’s life. Which of the following is not impacted by Pompe disease?
2. The accumulation of which molecule in the central nervous system leads to neuromuscular dysfunction associated with Pompe disease?
3. Which of the following does not contribute to the diagnostic delay experienced by people with LOPD?
4. Of the following, what is the most common cause of death for people with LOPD?

Meet the experts

Professor Benedikt Schoser 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 KishnaniDr. 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 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).

Welcome:

Developed independently by EPG Health, which received educational funding from Amicus Therapeutics, Inc. awarded to EPG Health to help provide its healthcare professional members with access to the highest quality medical and scientific information, education and associated relevant content.

In support of improving patient care, this activity has been planned and implemented by Amedco LLC and MEDTHORITY. Amedco LLC is jointly accredited by the Accreditation Council for Continuing Medical Education (ACCME), the Accreditation Council for Pharmacy Education (ACPE), and the American Nurses Credentialing Center (ANCC), to provide continuing education for the healthcare team.

Amedco Joint Accreditation #4008163.

Professions in scope for this activity are listed below.

Physicians
Amedco LLC designates this live activity for a maximum of 1.0 AMA PRA Category 1 CreditsTM. Physicians should claim only the credit commensurate with the extent of their participation in the activity.