Virtual Industry-Supported Symposia

• The Limb-Girdle Muscular Dystrophies - Jordi Díaz-Manera, MD, PhD
• Clinical Updates in Limb-Girdle Muscular Dystrophy 2E/R4 - Louise Rodino-Klapac, PhD
• Clinical Updates in Duchenne Muscular Dystrophy - Jerry Mendell, MD

16:00 - 17:00 CET

• 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

• 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

• 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

13:00 - 14:00 CET

• 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

16:00 - 17:00 CET

• 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

• 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: 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 and (Chair)