Industry Symposia

WMS Industry Symposium 1

The Era of Gene Therapy: Growing Evidence of The Clinical Reality

Sharing real-world evidence and practical guidance for SMA treatment with Gene Therapy! Our expert faculty will demonstrate the growing evidence of efficacy and safety outcomes following gene therapy administration, with a focus on bulbar function. We will discuss strategies to overcome challenges associated with gene therapy and our faculty will conclude the session by sharing practical guidance for managing patients receiving gene therapy. Audience members will also have the opportunity to ask our expert panel questions within a closing panel discussion. 

WMS Industry Symposium 2

Shared decision-making in the management of late-onset Pompe disease

Late-onset Pompe disease (LOPD) is characterized by variably progressive skeletal muscle dysfunction and respiratory insufficiency. Due to the multisystemic and heterogeneous nature of LOPD, individualized, comprehensive and holistic assessments help inform shared management decisions. 

In this cutting-edge symposium sponsored by Amicus Therapeutics, subject matter experts Dr Angela Genge (McGill University, Canada), Prof Kristl Claeys (University Hospitals Leuven, Belgium) and Prof Benedikt Schoser (Ludwig-Maximilians-Universität München, Germany) will be joined by Alex B. (an experienced advocate and person living with LOPD). Together, they will discuss the importance of shared decision-making in the management of LOPD and analyse traditional and investigational approaches that may be used to monitor signs of disease activity. Alex B. will also share a personal perspective on how LOPD can impact quality of life and everyday activities.

The event will close with a Q&A, with our expert panel answering your questions.

WMS Industry Symposium 3

Connecting the Dots: Charting New Frontiers in Duchenne

Join an expert-guided exploration of DMD from early diagnosis through disease heterogeneity followed by the utilization of natural history cohorts and disease modeling in clinical trials.

WMS Industry Symposium 4

Measuring Progression in FSHD: Implications for Clinical Trials

Every FSHD patient faces relentless and accumulating muscle and functional loss.  This symposium focuses on appropriate outcomes measures to assess the progression of FSHD in clinical trials, including Reachable Workspace (RWS) and Whole-Body Musculoskeletal MRI.  New results from the ongoing Phase 2 Open-Label Extension study of losmapimod, targeted to preserve muscle function in FSHD, will be discussed. 

WMS Industry Symposium 5

Moving the needle: improving outcomes in paediatric SMA and DMD

An expert panel chaired by Prof Eugenio Mercuri (Italy) will discuss progress in the management of paediatric SMA and advances in gene therapy for DMD. Key topics in the session will include the importance of early detection through newborn screening for SMA, progress in existing and emerging disease modifying treatments for SMA, an update on gene therapy trials in DMD, and clinically meaningful endpoints that address heterogeneity in evolving clinical DMD phenotypes.

WMS Industry Symposium 6

Rethinking the science of SMA

SMN restoring therapies are transforming patient outcomes in SMA. However, variability in treatment response at the individual level remains striking. We explore what is known about the genesis, degeneration and potential resurrection of the motor neuron in SMA, the host and intervention factors that may modify treatment response at the individual level, and the rationale to investigate strategies to optimise these responses.

WMS Industry Symposium 8

Navigating the changing disease landscape: continuity of care in Duchenne muscular dystrophy

Please note that the PTC Therapeutics symposium 8 has been rescheduled to this time from Friday morning. Lunch will be available during this session.

This symposium will showcase the latest clinical data from Study 041 and the STRIDE Registry; these studies offer promising results for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). The importance of early and continuous care throughout the disease continuum and how the multidisciplinary management of patients with DMD should evolve through disease milestones, will also be discussed.

WMS Industry Symposium 7

Targeting Fast Muscle Myosin: A Novel Approach to Protecting Muscle in the Dystrophinopathies

The symposium will explore how targeting fast muscle myosin could potentially protect muscle in Duchenne and Becker muscular dystrophy. The damaging consequences of muscle contraction without functional dystrophin will be presented by Dr. Alan Russell on behalf of Dr. Lee Sweeney.  Dr. John Vissing will discuss the impact of muscle contraction and exercise on biomarkers of fast muscle fiber damage in dystrophinopathies and other muscular dystrophies. Dr. Craig McDonald will discuss the clinical course of Becker muscular dystrophy and potential strategies for clinical trials. Dr. Alan Russell will discuss identification of a fast myosin modulator to disconnect muscle injury from muscle contraction in dystrophinopathies to circumvent the structural stress caused by loss of dystrophin in Duchenne and Becker muscular dystrophy. Lastly, Dr. Joanne Donovan will discuss most recent findings from clinical studies with EDG-5506 a novel, first-in-class, small molecule for the treatment of dystrophinopathies as well as other muscular dystrophies, including 6-month interim results from the ongoing ARCH study of EDG-5506 in adults with Becker muscular dystrophy.