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Tweets by ICNMD 2021

Friday, 11 September
Saturday, 12 September
Sunday, 13 September
Monday, 14 September
13:00 - 14:00 CET Gene Therapies for Neuromuscular Disorders
  • The Limb-Girdle Muscular Dystrophies - Jordi Díaz-Manera, MD, PhD (United Kingdom)
  • Clinical Updates in Limb-Girdle Muscular Dystrophy 2E/R4 - Louise Rodino-Klapac, PhD (USA)
  • Clinical Updates in Duchenne Muscular Dystrophy - Jerry Mendell, MD (USA)

For more details, see here.

16:00 - 17:00 CET

Complement Activation in Neurological Diseases: From the Central Nervous System to the Neuromuscular Junction
Chair: Dr. Angela Genge
  • Generalized myasthenia gravis: complement and the neuromuscular junction - Dr James F Howard, Jr (USA)
  • Neuromyelitis optica spectrum disorder: complement and astrocytes - Dr Dean Wingerchuk (USA)
  • Amyotrophic lateral sclerosis: complement and the spinal cord and neuromuscular junction - Dr Angela Genge (Canada)

For more details, see here.

13:00 - 14:00 CET

New Insights in the Use of Intravenous Immunoglobulins: How to Enhance CIDP Patients' Clinical Outcomes?
Chair: Eduardo Nobile-Orazio
  • Predicting the response to Intravenous Immunoglobulins in patients with CIDP - Helmar Lehmann, Germany
  • Individualized IVIg treatment as an optimization of therapeutic strategy in CIDP - Yusuf Rajabally, UK
  • A European efficacy and safety study of IVIg 10% in the treatment of patients with CIDP: The PRISM Study results - Eduardo Nobile-Orazio, Italy

For more details, see here.

16:00 - 17:00 CET

Pompe Disease: Neuromuscular and Respiratory Manifestations
  • Insights into the Phenotypes of Pompe Disease - Priya S. Kishnani, MD, Professor of Pediatrics and Division Chief, Medical Genetics, Professor of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC (USA)
  • Respiratory Involvement in Pompe Disease - Kenneth I. Berger, MD Professor of Medicine, Neuroscience and Physiology Division of Pulmonary, Critical Care, and Sleep Medicine, New York University School of Medicine, New York, NY (USA)
  • Q&A - Professors Kishnani and Berger

For more details, see here.

13:00 - 14:00 CET

Optimising Patient Care Across the Evolving SMA Landscape
Chair: Dr. Nathalie Goemans, Neuromuscular Reference Centre for Children, University Hospitals, Leuven, Belgium
  • Standard of care in SMA: real but not ideal - Dr. Nathalie Goemans (Chair), Neuromuscular Reference Centre for Children, University Hospitals, Leuven, Belgium
  • Getting prepared: emerging phenotypes in SMA - Dr. Mariacristina Scoto, Great Ormond Street Hospital for Children, London, UK, Tina Duong, Stanford University, Palo Alto, California, USA
  • Looking ahead: evidence-based treatment decision in SMA - Prof. Laurent Servais, MDUK Oxford Neuromuscular Centre, Oxford, UK
  • Panel Discussion with Q&A - all faculty
  • Summary and Symposium close - Dr. Nathalie Goemans (Chair), Neuromuscular Reference Centre for Children, University Hospitals, Leuven, Belgium

For more details, see here.

16:00 - 17:00 CET

Advancing the Management of Duchenne Muscular Dystrophy: the Role of Real-World Evidence
Chair: Professor Juan Vilchez, Valencia, Spain
  • Obtaining an accurate Duchenne muscular dystrophy (DMD) genetic diagnosis - Professor Annemieke Aartsma-Rus, Leiden, Netherlands
  • Examining real-world evidence for the treatment of nonsense mutation DMD - Professor Eugenio Mercuri, Rome, Italy
  • Q&A session - All speakers

For more details, see here.

13:00 - 14:00 CET

Time to SHINE on SMA: Exploring the Evidence from SHINE, the Nusinersen Open-Label Extension Study in Infants and Children
  • The nusinersen clinical development programme – Dr Angela Paradis, PhD, Biogen
  • Setting treatment expectations in infantile-onset SMA - Applying the evidence from ENDEAR-SHINE - Prof Francesco Muntoni, Great Ormond Street Hospital for Children, London, UK
  • Setting treatment expectations in later-onset SMA - Applying the evidence from CHERISH-SHINE - Dr Andrés Nascimento, Hospital Sant Joan de Déu, Barcelona, Spain
  • Q&A

For more details, see here.

16:00 - 17:00 CET

Past, Present and Future: Realizing the Potential of Gene Therapy for Neuromuscular Disorders
Chair: Dr Eduardo Tizzano
  • Past: A journey through the evolution of gene therapy - Prof Janbernd Kirschner
    - Brief overview of history of gene therapy
    - Principles and mechanism of action of gene therapy
    - Potential issues associated with gene therapy from a historical perspective
  • Present: Harnessing gene therapy for the treatment of NMDs - Dr Eduardo Tizzano
    - An introduction to NMDs including burden, prevalence and genetics
    - Current development of gene therapy for NMDs
    - Therapeutic window for NMDs and the importance of early diagnosis and intervention
  • Future: Navigating the clinical reality of gene therapy - Dr Samiah Al-Zaidy
    - Barriers for the translation of pre-clinical findings to the clinic
    - How will standard-of-care guidance evolve in light of gene therapy?
    - Questions that remain unanswered with future use of gene therapy including the management of emerging phenotypes
  • Q&A and Close - Dr Eduardo Tizzano (Chair)

For more details, see here.

The above-listed Virtual Industry-Supported Symposia will be aired “live” during ICNMDigital at the time listed, then available on-demand on the Virtual Library until ICNMD 2021 in Valencia.

In addition, the following symposium will also be available on-demand on the Virtual Library, after ICNMDigital has concluded.

Virtual Industry-Supported Symposium supported by UCB Biopharma
Myasthenia Gravis: Patient and Clinician Perspective
Chair: Prof. Pushpa Narayanaswami, USA

  • Short introduction - Prof. Pushpa Narayanaswami, USA
  • MG patient disease journey & unmet need from a patient perspective – Mr. Laurence F. Noonan Jr, USA
  • MG clinical evaluation and current limitations - Dr. Elena Cortes, Spain
  • Conclusion: what outcomes matter to patients living with MG and future research - Prof. Pushpa Narayanaswami, USA


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    The ICNMD is organized on behalf of the Applied Research Group on Neuromuscular Diseases of the World Federation of Neurology.
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