Industry symposia

WMS industry symposium 1 - Withdrawn

WMS industry symposium 2

Duvyzat: Advancing the treatment landscape in Duchenne muscular dystrophy

Chair: Laurent Servais
Speakers: Ulrike Schara-Schmidt, Erika Finanger

Opening and welcome by Dr. Laurent Servais (5 min)

Duvyzat: Advancing the treatment landscape for Duchenne Muscular Dystrophy, Dr. Schara-Schmidt (20 min)

Dr. Schara-Schmidt will explain why Duvyzat (givinostat) represents a novel approach to target Duchenne muscular dystrophy pathology and a significant advancement in the therapeutic landscape of Duchenne. She will continue sharing her experience with this new treatment recently available, providing guidance on patient management and practical recommendations about how DMD units should be organized. She will finalize sharing new data recently available like Duvyzat effects on cardiac function preservation.

Confirming durable efficacy and safety in DMD using long-term extension and natural history studies, Dr. Erika Finanger (20 min)

Dr. Erika Finanger will discuss the importance of natural history cohorts and long-term extension studies in diseases with progressive evolving courses like Duchenne muscular dystrophy. She will highlight the key learnings from natural history cohorts over time and the impact of standarized care. She will continue discussing the rational for open label long term extension studies to complement the information obtained from clinical trials ,and will finalize with an example showing a comparison between an open label extension study and existing Duchenne natural history cohorts.

Panel discussion moderated by Dr. Servais (15 min)

WMS industry symposium 3

Cardiomyopathy and advances in the treatment paradigm

Speaker: Kan Hor

Dr. Hor will share insights on why early detection and management of cardiomyopathy are crucial for improving the prognosis of DMD patients. There is currently no cure for DMD, but treatments focusing on managing symptoms and improving quality of life are leading research initiatives. Research is ongoing to develop new therapies that target the underlying cause of DMD and prevent or slow down the progression of cardiomyopathy. Characteristics identified by specific monitoring procedures, symptoms like cardiac fibrosis (scarring), arrhythmias (irregular heartbeats), and heart failure each which produce dilated cardiomyopathy and subsequent congestive heart failure. As stated earlier, this condition is the primary cause of death in patients with DMD.

WMS industry symposium 4

Achieving functional improvement in myotonic dystrophy type 1 (DM1): defining goals of treatment and a roadmap to multidisciplinary care

Speakers: Lori Planco, Monica Visone, Nicholas Johnson, Valeria Sansone

Myotonic dystrophy type 1 (DM1) is a spliceopathy that results in multisystemic involvement and significant disease burden. Individuals with DM1 experience a wide range of symptoms related to skeletal and non-skeletal muscle involvement, such as myotonia and muscle weakness, cardiac and pulmonary dysfunction, gastrointestinal issues, and CNS-related symptoms such as fatigue and cognitive impairment. The complexity of the disease mandates a comprehensive and coordinated multidisciplinary approach to care, with unique considerations for symptom management and prevention of complications. The goal of treatment should include therapeutic approaches that address the totality of symptoms in DM1.  

This symposium will feature an expert panel, including two adult neurologists and two DM1 community members, who will discuss the burden of disease and approaches to multisystemic disease management. Best practices and approaches to establishing an institutional multidisciplinary network to improve patient outcomes will also be discussed. Lastly, the symposium will explore the need to address the multisystemic nature of DM1 with therapeutic approaches that enable targeted delivery of disease-modifying therapies to address the totality of symptoms in DM1, including muscle- and CNS-related manifestations. The symposium will be interactive and will include a fireside chat, structured educational presentations, and audience engagement through an interactive Q&A.

WMS industry symposium 5

Evolving with SMA: A journey from survival to independence

Chair: Prof. Marcondes Franca
Speakers: Eugenio Mercuri, Tim Hagenacker

The treatment landscape in spinal muscular atrophy (SMA) is evolving, with patient expectations shifting from survival towards achieving greater independence. In this symposium, the speakers will explore how SMA treatment is reshaping functional expectations and the evolving phenotypic spectrum. This will include discussion of key unmet needs in paediatric and adult SMA, illustrated through real-world patient experiences that can help inform clinical practice.

WMS industry symposium 6

Breakthroughs in Becker: Unveiling New Natural History Insights and a Novel Agent’s Clinical Progress

Speakers: Roxana Donisa Dreghici, Erik Niks, Craig McDonald

Becker muscular dystrophy (Becker) is a serious rare disease with no currently approved treatment options. In this program, Erik Niks, MD, PhD, will present the latest natural history research in Becker and will discuss the factors that influence disease progression as supported by new prognostic modeling data. Craig McDonald, MD, will then present an overview of the clinical development of sevasemten, an investigational fast myosin inhibitor currently being studied in Becker, and share the latest results from CANYON, the largest interventional trial in Becker to date. He will also present emerging data from MESA, a long-term, open-label extension trial in adults with Becker. The session will close with a Q&A panel discussion with the audience.