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Get Tickets!

Industry-Supported Symposia

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The following industry-supported symposia, organized by ICNMD 2021 sponsors, were broadcast live during the 2021 Live Days. Click on the tabs below to read more.

You will find these symposia listed chronologically in the 'Symposia & Product Theatres' section of the virtual platform.

You may click directly on the dedicated symposium links below to be taken directly to each symposium page. You will need to log in with your platform credentials.

 

Friday, 21 May
Saturday, 22 May
Friday, 28 May
Saturday, 29 May
ISS01 - gMG 360: Generalised myasthenia gravis from cause to consequence
Time: 12:00 - 12:45 CEST
Agenda
  • 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

For more information, click here.
Click here for direct viewing.

ISS02 - A Novel Assessment of Swallowing in SMA: Insights from the NURTURE Study 
Time:
 12:00 - 12:20 CEST
Agenda
  • A Novel Assessment of Swallowing in SMA: Insights from the NURTURE Study
    Prof. Valeria Sansone, NEuroMuscular Omnicentre (NEMO), Italy
  • Live Q&A

For more information, click here.
Click here for direct viewing.

ISS03 - Current Treatment Landscape of Myotonia in Muscle Channelopathies: Focus on Mexiletine
Time: 12:25 - 12:45 CEST
Agenda
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

For more information, click here.
Click here for direct viewing.

ISS04 - Gene Therapy – Transforming the Clinical Outlook for Neuromuscular Diseases
Time: 17:15 - 18:00 CEST
Agenda
  • 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

For more information, click here.
Click here for direct viewing.

ISS05 - How to Overcome Challenges in Pompe Disease Management During the COVID-19 Era
Time: 17:15 - 18:00 CEST
Agenda
  • 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

For more information, click here.
Click here for direct viewing.

ISS06 - Intrahepatic Cholestasis in X-linked Myotubular Myopathy
Time: 12:00 - 12:45 CEST

Click here for direct viewing.

ISS07 - Meaningful Outcomes Across All SMA Types
Time: 12:00 - 12:45 CEST
Agenda
  • 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

For more information, click here.
Click here for direct viewing.

ISS08 - Serum Autoantibody Levels and the Role of Specificity in Myasthenia Gravis
Time: 16:15 - 17:00 CEST
Agenda
  • Introductions
    Dr Heinz Wiendl, Professor in Neurology and Neuroimmunology, Head of Department of Neurology and Translational Neurology, University of Muenster, Germany
  • MG Prevalence and Disease Burden
    Dr Heinz Wiendl
  • MG Pathophysiology as an IgG Autoantibody-Mediated Disease
    Dr Francesca Sacca, Assistant Professor in Neurology, University Federico II; Assistant Medical Director, AOU Federico II, Italy
  • The Effect of Current Therapies on AChR Autoantibody Reduction in Seropositive MG Patients
    Dr Pushpa Narayanaswami, Associate Professor of Neurology, Harvard Medical School; Vice Chair, Clinical Operations, Department of Neurology, Beth Israel Deaconess Medical Center, USA, and Dr Francesca Sacca
  • Investigational Agents Designed for IgG Autoantibody Depletion
    Dr Heinz Wiendl
  • Summary and Close
    Dr Heinz Wiendl
  • Live Q&A
    Dr Heinz Wiendl, Dr Francesca Sacca, Dr Pushpa Narayanaswami

For more information, click here.
Click here for direct viewing.

ISS09 - Investigating Gene Therapy for the Treatment of Limb Girdle Muscular Dystrophies
Time: 16:15 - 17:00 CEST
Agenda
  • Welcome and Introduction
    Dr. Louise Rodino-Klapac, Executive Vice President, Chief Scientific Officer, Sarepta Therapeutics Inc., USA
  • Identification and Diagnosis of LGMD Patients
    Dr. Jordi Díaz-Manera, Professor of Neuromuscular Diseases, Translational Medicine and Genetics, John Walton Muscular Dystrophy Research Centre, Newcastle University Translational and Clinical Research Institute, UK 
  • Limb Girdle Muscular Dystrophy Study 9003-101 Interim Findings
    Dr. Erica Koenig, Director, Clinical Development, Sarepta Therapeutics Inc., USA 
  • Closing Remarks & Q&A
    All Faculty

For more information, click here.
Click here for direct viewing.

ISS10 - Can We Regain Control? A Clinical Case-Based Discussion on the Treatment of Refractory Generalized Myasthenia Gravis
Time: 12:00 - 12:45 CEST
Agenda
  • 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

For more information, click here.
Click here for direct viewing.

ISS11 - A Unique Pregnancy Case: Venturing Beyond the Norms in Pompe Disease
Time: 12:00 - 12:20 CEST
Agenda

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

For more information, click here.
Click here for direct viewing.

ISS12 - What Matters? Setting Treatment Expectations for Motor Function in SMA Type 1
Time: 12:25 - 12:45 CEST
Agenda
  • What Matters? Setting Treatment Expectations for Motor Function 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

For more information, click here.
Click here for direct viewing.

ISS13 - Understanding the Immunomodulatory Effects and Clinical Benefits of IVIG in Myasthenia Gravis
Time: 16:15 - 17:00 CEST
Agenda
  • 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

For more information, click here.
Click here for direct viewing.

ISS14 - Advancing the Management of Duchenne Muscular Dystrophy: The Disease Continuum and Transition from Pediatric to Adult Care
Time: 16:15 - 17:00 CEST
Agenda
  • 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)

For more information, click here.
Click here for direct viewing.

ISS15 - Life and Death in the Neuron: the Role of the ER and Mitochondria in ALS
Time: 12:00 - 12:45 CEST
Agenda

Speakers: Stephanie Hoffmann, Acting Head of EUMEA, Amylyx Pharmaceuticals (Chair)
Prof Adriano Chio, Professor of Neurology and Director, ALS Center, University of Torino, Italy

  • 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

For more information, click here.
Click here for direct viewing.

ISS17 - Suspecting, Diagnosing, and Managing Multifocal Motor Neuropathy: A Patient Investigation
Time: 17:15 - 18:00 CEST
Agenda
  • 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

For more information, click here.
Click here for direct viewing.

ISS18 - Gene Transfer Therapy: Addressing the Unmet Needs of Duchenne Muscular Dystrophy (DMD)
Time: 17:15 - 17:35 CEST
Agenda

Speaker: Professor Wolfgang Müller-Felber, Dr von Hauner Children’s Hospital, Germany
  • 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

For more information, click here.
Click here for direct viewing.


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