Industry Symposia

WMS Industry Symposium 1

From Innovation to Patient Empowerment: The Pivotal Role of Complement
Speakers: Benedikt Schoser, Vasiliki Zouvelou

A 60-minute symposium comprising 2 presentations and 15-minute Q&A session.

The first presentation will be delivered by Dr. med. Benedikt Schoser and will detail the burden of disease in gMG and the role of complement in the pathophysiology, overview of the clinical value of C5 inhibition using trial data from CHAMPION-MG and Recent MG treatment guidelines, including where C5 inhibitors fit into the therapeutic landscape. Finally, the session will finish with a review of the steroid-sparing effect of C5 inhibition.

In the second session Dr. Vasiliki Zouvelou will present how the patients should be prioritized and Patient-Centric Approach to Generalized Myasthenia Gravis Treatment will be discussed.

WMS Industry Symposium 2

Understanding disease progression of Becker Muscular Dystrophy and a potential novel agent to protect muscle
Moderator: Joanne Donovan,
Speakers: Luca Bello, Craig McDonald,

Becker muscular dystrophy is a serious rare disease with no specific treatment options. In this program, Luca Bello, MD, PhD, will present recent updates from the largest natural history study in Becker, with over 5 years of follow-up, and how they add to the landscape of previously published natural history studies. He will then discuss how these studies inform the selection of clinical outcome measures and clinical characteristics in the design of future clinical trials in Becker. These data provide additional perspective on prognosis for individuals with Becker, highlighting that once functional decline begins, individuals with Becker continue on a trajectory to irreversible muscle loss. Craig M. McDonald, MD, will provide a critical overview of outcome assessments in Becker that can be utilized to assess disease progression and their clinical meaningfulness. He will share recent updates from Becker clinical trials utilizing these outcome measures, including an overview of the clinical development of sevasemten, a novel fast myosin inhibitor, which is currently being investigated in Becker with an actively recruiting pivotal trial. The session with close with Q&A panel discussion with the audience.

WMS Industry Symposium 3

Assessing and managing DMD in an evolving treatment landscape
Chair: Laurent Servais
Speaker: Ulrike Schara-Schmidt

With the development of new therapies in Duchenne muscular dystrophy (DMD), we are witnessing an evolution of disease management. This symposium presentation will include an expert panel of clinicians and focus on the natural history and disease progression in DMD (including the pathogenic mechanisms), current standard of care, and recent advances within the treatment landscape. Other topics to be discussed include the importance of functional assessment tools currently used in DMD,
challenges of assessing, managing, and monitoring patients with DMD, and clinical implications of new treatments for patients, caregivers and the wider multidisciplinary team.

WMS Industry Symposium 4

Gene Therapy Beyond Borders: A Global Exploration of Today’s Achievements and Unmet Needs, and Tomorrow’s Uncharted Territories
Symposium Chair: Jana Haberlová
Speakers: Eugenio Mercuri, Rodrigo Mendonça, Vivek Mundada

In this symposium, we will review learnings from gene therapy in SMA to build an understanding of diverse approaches to treating neuromuscular disease worldwide, and address unanswered questions from patients, families, and the community.

• First, we will discuss evidence across clinical trials, real-world studies, and patient cases to provide an understanding of gene therapy for patients with SMA, including populations not extensively studied. We will discuss evidence from heterogenous countries where access to health systems vary, referral for diseases such as SMA are rare, and data describing compliance to standard-of-care recommendations are limited.
• Second, we will address unanswered questions surrounding newborn screening, diagnosis, treatment selection, long-term outcomes, and multidisciplinary care of patients with SMA that receive gene therapy.
• Finally, we will discuss learnings from SMA gene therapy and its relevance to other emerging treatments for neuromuscular disease. The global speaker panel will provide first-hand lessons learned from treating SMA, practical treatment considerations, and future approaches for neuromuscular disease.

This is a promotional meeting organized and funded by Novartis Pharma AG for healthcare professionals only and will include discussion of Novartis products.

WMS Industry Symposium 5

Time Matters: Preserving Function for Patients With Duchenne Muscular Dystrophy
Moderator: Francesco Muntoni
Speaker: Ros Quinlivan

This symposium will be an expert-led discussion on the management of Duchenne muscular dystrophy (DMD) from early to advanced stages of disease. This presentation will explore the natural history of disease progress, highlight the goal of functional preservation and dystrophin restoration.

WMS Industry Symposium 6

Impact of disease-modifying therapies on bulbar function in children and adults living with SMA: Learnings and limitations
Chair: Giovanni Baranello
Speakers: Katlyn McGrattan, Giorgia Coratti

At WMS 2023, the Roche-sponsored symposium explored the importance of bulbar function in spinal muscular atrophy (SMA). At WMS 2024, the same expert faculty will explore the impact of disease-modifying therapies on bulbar function in children and adults living with SMA and share their experiences of case management, with a focus on best practices in relation to the assessment, treatment and rehabilitation of bulbar function.

WMS Industry Symposium 7

Potential of the FORCE™ Platform to Address Unmet Needs in Rare Neuromuscular Diseases
John Vissing, Michela Guglieri, James Lilleker

The Dyne FORCE platform was developed to overcome limitations of oligonucleotide delivery to muscle for the treatment of rare neuromuscular disorders.

This symposium will provide an overview of how the FORCE platform leverages the natural biology of the cell for targeted delivery of oligonucleotides to muscle, with the goal of addressing the underlying cause of disease. Clinical data demonstrating the impact of the FORCE platform on key biomarkers and multiple clinical endpoints in myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) will be presented, with an overview of its application in other neuromuscular disorders.

WMS Industry Symposium 8

Emerging endpoints in Duchenne muscular dystrophy: From clinical trials to clinical practice
Chair: Odile Boespflug -Tanguy
Speakers: Astrid Blaschek, Han Phan

This symposium will focus on advancements in assessing treatment outcomes in Duchenne Muscular Dystrophy (DMD), highlighting clinical outcomes, innovative endpoints, and new approaches to clinical trials. Experts will discuss the progressive nature of DMD, functional assessments, and advanced imaging techniques as biomarkers.

Key Presentations:

1. Astrid Blaschek from Germany plans to discuss the evolution of clinical outcomes assessment in DMD. She will explain the progressive loss of physical function in DMD patients, the natural history of the disease, and how function is assessed over time. Various timed function tests, such as the four-stair climb, TTR - time- to- rise, and North Star Ambulatory Assessment will be analyzed, emphasizing their relevance for different age and functional groups.
2. Han Phan from the USA plans to cover new approaches in DMD disease assessment, focusing on clinical endpoints. She will discuss the innovative DMD clinical trial design in terms of patient stratification, study outcomes, dosing, and monitoring. She will also address the role of skeletal muscle MRI and MRS as biomarkers.

The event will conclude with a round table discussion moderated by Odile Boespflug-Tanguy from France, allowing for an in-depth discussion of the presentations and considering the questions from the audience.

This symposium will offer a comprehensive overview of current strategies in DMD assessment and patient management, emphasizing the integration of new biomarkers and functional endpoints into clinical practice.