Industry-Supported Symposia - ICNMDigital 2023
The following Industry-Supported Symposia are organized by sponsors of ICNMDigital 2023.
Click on the “Day 2” tab to view the two symposia on Friday 1 December.
Scroll down for information on an exclusive Product Theatre presentation.
ISS 1
Time: 3:15 pm – 4:15 pm CET
Title: From Clinical Trials to Clinical Practice: What Have We Learnt About Zolgensma (Onasemnogene Abeparvovec) Treatment in SMA?
Description
This is a 1-hour promotional symposium, organised and funded by Novartis Gene Therapies for healthcare professionals only. This presentation will include discussion of the Novartis Gene Therapies product onasemnogene abeparvovec (Zolgensma).
Chair and speakers:
• Dr Sean Wallace Pediatric Neurologist; MD, PhD Department of Clinical Neurosciences for Children, Oslo University Hospital, Norway (Chair)
• Prof. Eugenio Mercuri Professor of Paediatric Neurology; MD, PhD Gemelli University Hospital, Italy
• Dr Hugh McMillan Professor, Pediatric Neurology; MD, MSc, FRCPC Children’s Hospital of Eastern Ontario, Canada
SMA: From Diagnosis to Long-Term Care
• Sean Wallace (Norway)
Long-Term Outcomes of Patients Treated with Zolgensma
• Eugenio Mercuri (Italy)
What Does RWE Tell Us About Zolgensma Treatment for Children With SMA?
• Eugenio Mercuri (Italy)
Zolgensma Treatment Benefit: Risk in a Broader Patient Population?
• Hugh McMillan (Canada)
Direct link to symposium virtual page: click here (for registered delegates only; must be logged in to the platform first; platform going live on November 28)
For more details, click on Description or here.
ISS 2
Time: 3:15 pm – 4:15 pm CET
Title: Navigating the Changing Disease Landscape: Continuity of Care and the Totality of Evidence in nmDMD
Description
Join us to learn more about the totality of evidence for a treatment for nonsense mutation Duchenne muscular dystrophy, and the challlenges faced by patients transitioning from paediatric to adult care.
Agenda:
• Welcome
• Totality of evidence for a treatment in patients with nmDMD
• Real-world data for a treatment in patients with nmDMD
• Transitioning from paediatric to adult care for patients with nmDMD
• Panel discussion and closing
Speakers:
• Prof. Alexandra Prufer, Associate Professor of Pediatric Neurology, Department of Pediatrics, The Federal University of Rio de Janeiro, Brazil
• Prof. Luca Bello, Associate Professor of Neurosciences, University of Padova, Italy
• Prof. Rosaline Quinlivan, Professor of Neuromuscular Disease, MRC Centre for Neuromuscular Disease, National Hospital for Neurology and Neurosurgery, London, United Kingdom
• Dr. med. Christian Werner, Executive Director, Global Medical Affairs, Global DMD Lead, PTC Therapeutics (Chair)
Direct link to symposium virtual page: click here (for registered delegates only; must be logged in to the platform first; platform going live on November 28)
ISS 3
Time: 6:45 pm – 7:45 pm CET
Title: Limb-Girdle Muscular Dystrophies: Clinical Course of Disease and Advances in Gene Therapy Development
Description:
This program aims to review the current understanding and unmet needs of limb-girdle muscular dystrophies (LGMDs) as well as explore the rationale and clinical data for an investigational gene therapy for a particular LGMD subtype, LGMD2E/R4.
Agenda:
• Welcome & Introductions
• The Clinical Course: Understanding LGMD Disease State and Unmet Needs
• Evolving the Approach: Investigational SRP-9003 for LGMD2E/R4
• Q&A
Speakers:
• Dr. med. André Müller-York, Senior Director, LGMD Medical Lead, Global Medical Affairs, Sarepta Therapeutics, Inc.
• Jesantha Coy, B.Pharm, Executive Director, Global Program Team Leader, Sarepta Therapeutics, Inc.
Live only; no on-demand
Direct link to symposium virtual page: click here (for registered delegates only; must be logged in to the platform first; platform going live on November 28)
For more details, click on Description or here.
ISS 5
Time: 3:15 pm – 4:15 pm CET
Title: Aging and Infectious Diseases in Myasthenia Gravis
Description:
Chair and speakers:
• Prof. Vera Bril, University of Toronto, Toronto, Ontario, Canada
• Prof. Nils Erik Gilhus, University of Bergen, Bergen, Norway
Introduction (5 minutes)
• Prof. Vera Bril, University of Toronto, Toronto, Ontario, Canada
Aging in Myasthenia Gravis (20 minutes)
The aim of this presentation is to review current epidemiologic trends in myasthenia gravis (MG) and discuss MG subtypes based on onset age, with a focus on clinical characteristics and outcomes of very-late-onset MG.
• Prof. Vera Bril, University of Toronto, Toronto, Ontario, Canada
Myasthenia Gravis and Infectious Diseases (20 minutes)
The aim of this presentation is to review the complexities of infections in MG patients and discuss risk for infections as well as prophylactic and curative treatment.
• Prof. Nils Erik Gilhus, University of Bergen, Bergen, Norway
Q&A and wrap up (15 minutes)
• Prof. Nils Erik Gilhus
• Prof. Vera Bril
Direct link to symposium virtual page: click here (for registered delegates only; must be logged in to the platform first; platform going live on November 28)
ISS 6
Time: 3:15 pm – 4:15 pm CET
Title: New Strategies for Advancing Care and Protecting Bone Health in Duchenne Muscular Dystrophy
Description:
Chair: Prof Eugenio Mercuri, Catholic University, Rome, Italy
Where are we now? A review of current best practice for Duchenne muscular dystrophy (20 minutes)
• Prof Eugenio Mercuri, Catholic University, Rome, Italy
Vamorolone for Duchenne muscular dystrophy: current evidence and future development (20 minutes)
• Dr Michela Guglieri, Newcastle University, UK
What is the long-term impact of corticosteroids on bone health in Duchenne muscular dystrophy? (20 minutes)
• Dr Leanne Ward, University of Ottawa, Canada
Live only; no on-demand
Direct link to symposium virtual page: click here (for registered delegates only; must be logged in to the platform first; platform going live on November 28)
For more details, click on Description or here.
Product Theatre Presentation
In addition to the above live-streamed symposia, the following presentation will be available for on-demand consumption during ICNMDigital 2023 and after, starting Tuesday 28 November all the way through October 2024.
Making every moment count for boys with nonsense mutation Duchenne muscular dystrophy (nmDMD)
Join PTC Therapeutics to learn more about the totality of evidence for a treatment for nonsense mutation Duchenne muscular dystrophy.
For any questions, please email at medinfo@ptcbio.com.
Direct link to product theatre virtual page: click here (for registered delegates only; must be logged in to the platform first; platform going live on November 28)