Industry-Supported Symposia

• Introduction
  Prof. John Vissing, Professor of Neurology; Director, Neuromuscular Clinic and Research Unit, Department of Neurology, University of Copenhagen, Denmark (Chair)
• Initial outlook: The immune system in rare autoimmune neuromuscular diseases
  Prof. Heinz Wiendl, Head of Department, Chair, Department of Neurology, Institute of Translational Neurology, University Hospital Muenster, Germany
• Full circle: Clinical consequences of gMG
  Prof. John Vissing
• Q&A
  All
• Closing the loop
  Prof. John Vissing

• A Novel Assessment of Swallowing in SMA: Insights from the NURTURE Study
  Prof. Valeria Sansone, NEuroMuscular Omnicentre (NEMO), Italy
• Live Q&A

Speaker: Prof Savine Vicart, University Hospital Pitié-Salpêtrière, France

• Overview on unmet medical needs in non-dystrophic myotonia, NDM
• Phase 3 clinical trial outcomes, MYOMEX Study
• NaMuscla (mexiletine) is an approved anti-myotonic in NDM
• Summary and challenges in NDM management

• Welcome & Introduction – Challenges of Early Treatment in Neuromuscular Diseases
  Prof Laurent Servais, Professor of Paediatric Neuromuscular Diseases – MDUK Oxford Neuromuscular Centre, UK, and Invited Professor of Child Neurology, Liège University, Belgium
• Potential of Gene Therapy for Neuromuscular Diseases
  Dr Janbernd Kirschner, Department of Neuropediatrics, University Hospital Bonn, Germany
• Capturing the Clinical Perspective on Gene Therapy
  Sandra P. Reyna, VP, Global Medical Affairs, Novartis Gene Therapies, USA (Moderator)
  Prof Laurent Servais and Dr Janbernd Kirschner
• Closing & Q&A
  Sandra P. Reyna

• Welcome
  Dr. Stephan Wenninger (Chair)
• Optimizing Treatment of Pompe Disease
  Dr. Stephan Wenninger
• A Local Perspective
  Dr. Marcelo Rugiero
• Q&A
  Dr. Stephan Wenninger (Chair) + Dr. Marcelo Rugiero

• Overview of XLMTM
  Dr Weston Miller, Senior Medical Director, Astellas Gene Therapies, USA
• Presentation of four cases of intrahepatic cholestasis in untreated XLMTM patients
  Dr Cristina Molera, Servicio de Gastroenterologia, Hepatologia i Nutrició Pediàtrica, Hospital Sant Joan de Deu, Spain
• Q&A

• Welcome and Introduction
  Prof. Laurent Servais (Chair) 
• A New Chapter: Beyond Motor Function in Type 1 SMA
  Prof. Laurent Servais + Panel discussion 
• Q&A
  All faculty 
• Changing the Narrative: What is Meaningful in Type 2 or 3 SMA?
  Prof. Anna Kostera-Pruszczyk + Panel discussion 
• Q&A
  All faculty 
• Summary and Close
  Prof. Laurent Servais

• Welcome and introductions
  Dr Carlos Casasnovas, Hospital de Bellvitge, Spain (Chair)
• Impact of refractory generalized myasthenia gravis on patient well-being
  Dr Carlos Casasnovas, Hospital de Bellvitge, Spain
• Complement in the pathophysiology and treatment of refractory generalized myasthenia gravis
  Dr Nico Melzer, Universitätsklinikum Düsseldorf, Germany
• Real-world experience with refractory generalized myasthenia gravis
  Dr Hiroyuki Murai, International University of Health and Welfare, Japan
• Questions and close
  Chair and speakers

Speakers: Prof. Priya Kishnani, Professor of Pediatrics, Duke Department of Pediatrics, USA (chair & speaker) 
Dr. Sheela Nampoothiri, Clinical Professor & Head of Department of Pediatric Genetics, Amrita Hospital, India (speaker)

• Welcome
• Joint Discussion of a unique pregnancy patient case study and its related challenges and impacts in managing Pompe disease
• Q&A

• What Matters? Setting Treatment Expectations in SMA Type 1
  Prof. Laurent Servais, Muscular Dystrophy UK (MDUK), MDUK Oxford Neuromuscular Centre, University of Oxford, UK and University of Liege, Belgium
• Live Q&A

• Introduction
  Prof. Dr. Marinos C. Dalakas (chair), Thomas Jefferson University, USA
• The Role of IVIG in the Immunomodulation of Myasthenia Gravis
  Prof. Dr. Marinos C. Dalakas, Thomas Jefferson University, USA
• New Data on IVIG Treatment of Myasthenia Gravis
  Prof. Dr. Andreas Meisel, Integrated Center for Myasthenia Gravis, Charité – University Medicine Berlin, Germany  
• Q&A

• Welcome and Introduction
  Dr. med. Christian Werner, Executive Director, Global Medical Affairs – Global DMD Lead, PTC Therapeutics (Chair)
• DMD as a Disease Continuum: a Succession of Management Milestones
  Prof Tracey Willis, Consultant Paediatric Neurologist, The Robert Jones and Agnes Hunt Orthopaedic Hospital, England
• The Transition from Pediatric to Adult Care for Patients with nmDMD
  Prof Thomas Sejersen, Professor in Neuropediatrics, Karolinska University Hospital and Karolinska Institute, Sweden
• Panel Discussion and Q&A: Managing Patients with nmDMD throughout the Disease Continuum
  Moderated by Dr. med. Christian Werner (Chair)

• The role and common dysfunctions of ER and mitochondria in the neurodegenerative disorders, including ALS
• The multiple complex pathways in ALS pathophysiology and how they lead to neurodegeneration
• The rationale for a multipronged approach simultaneously targeting multiple pathways underlying ALS
• Live Q&A

• Welcome and Introduction
  Dr. Mark B. Bromberg, University of Utah, USA 
• To Diagnose You First Have to Suspect: Establishing the Early Clues of MMN
  Dr. H. Stephan Goedee, University Medical Center Utrecht, The Netherlands 
• The Mystery of the Pathogenesis of MMN: What Do We Know?
  Dr. Mark B. Bromberg
• New Developments in Ig Therapy for MMN: What Matters to Patients
  Dr. Ali Al-Zuhairy, Copenhagen University Hospital, Denmark 
• “Ask the Faculty” and Take-Home Messages
  Dr. Mark B. Bromberg, Dr. H. Stephan Goedee, and Dr. Ali Al-Zuhairy

• Key Milestones in the History of Gene Therapy
• An Overview of Gene Therapy
• Gene Transfer Therapy to Treat DMD: Rationale and Challenges
• Gene Therapy, a Once-in-a-Lifetime Treatment?
• Summary and Close