Industry-Supported Symposia
The following Industry-Supported Symposia are organized by sponsors of ICNMD 2024. More Details will be provided in the next few months.
- ISS01
- Title: Treatment Approaches for Pre-Symptomatic Infants Living with SMA
- Time: 13:00 – 14:00
- Room: Riverview Room 5
- Topic: SMA
- ISS02
- Title: To the Joy of Options - Exploring Targeted Treatment Approaches in gMG
- Time: 13:00 – 14:00
- Room: M1
- Topic: MG
How do the mechanisms of action differ between gMG treatments and what is the associated immunological impact?
How have targeted therapies been shown to improve outcomes in adult patients with gMG?
How can targeted treatment options help to support the specific needs of patients and enhance treatment plans?
Speakers
Associate Professor Stephen Reddel
Dr Ali A. Habib
Professor James F. Howard, Jr
- ISS03
- Title: Immunoglobulin Therapies in Practice: Overcoming Challenges Along the CIDP Patient Journey
- Time: 13:00 – 14:00
- Room: Riverview Room 4
- Topic: CIDP
Following an introduction to the pathology of CIDP, the faculty will present patient cases based on their clinical experience and explore the challenges of CIDP management.
The symposium will follow a discussion-style format with interactive audience engagement, focusing on strategies to overcome challenges, enhance clinical outcomes and improve patient care.
Key Objectives
– Educate on the pathology of CIDP
– Explore how understanding the pathology of CIDP informs management strategies
– Learn from real-world experiences on how to overcome challenges in the management of CIDP to enhance clinical outcomes and patient care
- ISS04
- Title: Advancing Neurodegenerative Research: Mechanisms, Biomarkers & Treatment Challenges
- Time: 07:00 – 08:00
- Room: M1
- Topic: SMA
Biogen will bring together a panel of leading international experts in neurological andneurodegenerative diseases to discuss opportunities in the treatment of spinal muscular atrophy, amyotrophic lateral sclerosis and Friedreich ataxia, including novel therapeutic targets and biomarkers.
Speakers
Andrew Corbett
Prof. Michelle Farrar
Prof. Martin Delatycki
Prof. Matthew Kiernan
Dr. Sandi Kariyawasam
- ISS05
- Title: Breaking New Ground In The Treatment Of Guillain-Barré Syndrome
- Time: 07:00 – 08:00
- Room: M8
- Topic: TBA
Guillain-Barré syndrome (GBS) is an autoimmunedisorder where an infection triggers IgM and IgG antibodies that cross-react with gangliosides in peripheral nerve components, activating C1q and the classical complement pathway. Understanding the role of complement and biomarkers in GBS pathogenesis provides viable targets for treatment. Immunotherapy that can block the entire classical complement pathway to target complement-mediated neuroinflammation and nerve damage is needed.
Symposium Objectives
- Address the risk of GBS as a serious life-threatening neurological emergency that requires early diagnosis and targeted treatment
- Review the role of classical complement activity as a key mediator of nerve fiber damage and nerve fiber repair in GBS
- Present data on the potential role of inhibiting C1q to improve the overall health status of patients with GBS
Speakers
Mazen Dimachkie, MD
Henk-André Kroon, MD, MBA
Jeff A. Allen, MD
- ISS06
- Title: Spotlight on nmDMD: Exploring a Targeted Treatment Option
- Time: 13:00 – 14:00
- Room: Riverview Room 4
- Topic: DMD
Symposium Objectives
• Learn about the development history of ataluren* for the treatment of patients with nonsense
mutation Duchenne muscular dystrophy (nmDMD)
• Review the data on ataluren* treatment in patients with nmDMD
• Discuss the importance of early and continuous treatment throughout the disease
continuum to maximize the quality of care for patients with nmDMD
Speakers
Dr Maina Kava
Dr Eppie Yiu
Prof Francesco Muntoni
- ISS07
- Title: Treating Myasthenia Gravis by Targeting FcRn: Clinical Data and Perspectives From Global Experts
- Time: 13:00 – 14:00
- Room: Riverview Room 5
- Topic: MG
Join the conversation as leading international experts present and discuss generalized myasthenia gravis, including
• An overview of pathophysiology and treatment landscape
• The role of FcRn inhibition in treating AChR-Ab+ gMG
• A patient case with clinical perspectives from around the world
Speakers
James F. Howard, MD, FAAN
Dr. Barnett-Tapia, MA, PhD
Hiroyuki Murai, MD, PhD
- ISS08
- Title: Reimagining SMA Care with Newborn Screening and Gene Therapy
- Time: 13:00 – 14:00
- Room: M1
- Topic: SMA
Considering the recent advancements in SMA management, the symposium will examine the impact of gene therapy on treatment outcomes utilizing insights from the implementation of newborn screening.
Speakers
Prof Michelle Farrar (Symposium Chairperson)
Dr Richard Finkel (International speaker)
Dr Didu (Sandi) Kariyawasam (Australian speaker)
- ISS09
- Title: Advancements in CIDP: The Clinical Updates From Recent argenx Trials
- Time: 13:00 – 14:00
- Room: Riverview Room 4
- Topic: CIDP
Join our expert speakers as they share insights onvarious aspects of chronic inflammatory demyelinating polyneuropathy (CIDP) and its treatment, including:
• The patient journey in CIDP and updates from recent argenx clinical trials
• Clinical overview and pathophysiology of CIDP
• A brief overview of clinical tools used in assessing patients with CIDP
• Role of neonatal Fc receptor (FcRn) inhibition
- ISS10
- Title: Preparing for A Future with Gene Therapy in DMD: A Journey Towards Inclusion and Equity
- Time: 13:00 – 14:00
- Room: Riverview Room 5
- Topic: DMD
Join us at this symposium to discuss the advances in DMD gene therapies with a spotlight on health equity!
- Explore the practical and ethical challenges around introducing gene therapy as a potential treatment option for DMD
- Examine the available clinical data for DMD gene therapies in the context of disease progression, outcome measures and safety signals
- Highlight important considerations to overcome identified challenges associated with gene therapy delivery
- Time: 13:00 – 14:00
- Topic: FSHD
- ISS11
- Title: Navigating the Disease Journey: Pathophysiology and Progression of LGMDs
- Time: 13:00 – 14:00
- Room: Riverview Room 5
- Topic: LGMD
Sarepta’s spotlight session at ICNMD will discuss key aspects of limb-girdle muscular dystrophies (LGMDs) for diagnosis and monitoring of disease progression. This presentation will examine the pathology of LGMDs, highlighting the importance of accurate diagnosis, exploring the crucial role of natural history studies, and reviewing markers of disease progression.
Speakers
Volker Straub, MD, PhD
Ian Woodcock, MBBS, PhD, MSc, FRACP
Meredith James, PhD, PT
- ISS12
- Title: Treatment of Generalized Myasthenia Gravis (gMG)
- Time: 13:00 – 14:00
- Room: Riverview Room 4
- Topic: MG
Learning objectives
– Explore the mode of action and clinical development of nipocalimab in gMG.
– Discuss and reflect on the topline results from the pivotal phase III VIVACITY study of nipocalimab
Speakers
A/Prof. Stephen Reddel
A/Prof. Jin-Sung Park