A Novel Approach to Protecting Dystrophic Muscle by Targeting Fast Muscle Myosin: EDG-5506
A Treatment Option for Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
Rayne Rodgers, MPH (Co-Chair)
Elisabeth Kilroy, PhD (Co-Chair)
Session will discuss real-world data in neuromuscular disease and its application to therapy development.
Supporting Patients with SPINRAZA ® (nusinersen): Learn More About Our Clinical Trial Program and the Latest Real-World Evidence
Innovation for Rare Neuromuscular Diseases
What Lies Beneath: Uncovering the Clue for Earlier Diagnosis and Care of Young Individuals with Duchenne
Nicolas Johnson, MD, MS-CI (Chair)
Session will review advances in translational research in LGMD. Speakers will discuss steps to improve genetic diagnosis, development of small molecules, and advances in gene replacement therapy.
(CME Accredited Session)
Alan Beggs, PhD (Chair)
Angela Lek, PhD (Co-Chair)
Session will provide an overview of the rich and rapidly evolving body of knowledge in this area, with talks focused on collaborative and crowd-sourcing approaches to information sharing for new disease gene discovery, the latest on genetic heterogeneity among inherited peripheral neuropathies, approaches to undiagnosed neuromuscular diseases, and new genetic modifiers, which can impact the course of a patient’s disease.
Jeffrey Statland, MD (Chair)
Session explores implementation of targeted therapies in clinical trials, our molecular understanding of FSHD, and how we measure changes in future clinical trials
Henry Kaminski, MD (Chair)
Session will review the journey of treatment to patient from identification of unmet need to prescription of the drug and access for all patients.
(CME Accredited Session)
Dr. Erik Henricson, PhD, MPH (Chair)
Session will explore the uses and intersections between Clinically Reported Outcome tools, Person-Reported Outcome tools, and single- and multi-domain composite endpoints in DMD clinical research. Emphasis will be placed on how tools create a continuum of measures that can be extended from observed clinical task performance, to individual perceptions of ability, to community mobility and participation and life satisfaction
Chamindra Konersman, MD (Chair)
Session will provide understanding of the disease spectrum, genetics and phenotypic complexity of congenital myopathies and congenital muscular dystrophies. Understand current and future strategies for disease modifying treatments and preparing for meaningful clinical trial outcome measures.
(CME Accredited Session)
Brad Troxler, MD (Chair)
Session will provide a forum to learn about the latest advancements in respiratory care for patients with NMD. Use of advanced airway clearance techniques and positive airway pressure devices will be discussed. The impact of respiratory care on patient with NMD will be shared.
Amyotrophic Lateral Sclerosis (ALS) in Depth: Key Topics in Pathophysiology and Diagnosis
Measuring Impact on Disease Progression in FSHD
Carlos Moraes, PhD (Chair)
Session will explore a few approaches with great clinical potential that use small molecules or genetic manipulation.
Susan Apkon, MD (Chair)
Session will provide a greater understanding of the barriers to participation in clinical research for patients from diverse backgrounds and opportunities to improve their involvement. Panelists will share their diverse experience and perspectives as clinicians, family members, and industry partners.
(CME Accredited Session)
Dustin Nowacek, MD (Chair)
Session will discuss telehealth experiences, identify the benefits of telehealth, and recognize the limitations of virtual care
(CME Accredited Session)
John Brandsema, MD (Chair)
Session aims to review cases from neuromuscular practice that have been complicated ethically and generate an open forum for discussion of how to best navigate similar situations when they arise
Helen Blau, PhD (Co-Chair)
Michael Rudnicki, OC, PhD, FRS, FRSC (Co-Chair)
Session will discuss small molecule drugs that counter muscle loss and show promise in treating SMA, DMD, and BMD.
James Berry, MD, MPH (Chair)
Session will explore digital outcomes measures in neuromuscular disease therapy development
(CME Accredited Session)
Michael Shy, MD (Chair)
Session will provide an update on natural history studies, a description of pediatric patient-reported outcomes assessment evaluating the self-reported quality of life for children with CMT, approaches to evaluating and managing children with CMT, and approaches in clinical trial design in children with CMT and other rare forms of NMD
(CME Accredited Session)
Lora Clawson, MSN, CRNP (Chair)
Session will provide an overview of MDA clinical programs & collaborators; describe how these collaborators are engaged; and describe the challenges and successes in maintaining these important collaborations.
Explore the Science for an Approved Spinal Muscular Atrophy Treatment and Hear from People Living with SMA
Throughout the Disease Continuum: Corticosteroid Treatment in Duchenne Muscular Dystrophy
The Intersection of Science and Medicine – An Investigational rAAVrh74-based Gene Transfer Therapy for Individuals with Duchenne
(CME Accredited Session)
Yaacov Anziska, MD (Chair)
Session will address experiences from a range of specialists about coordinating with the neurologist to provide the best care for our MDA neuromuscular patients
(CME Accredited Session)
Teepu Siddique, MD (Chair)
Daniel Rubin, MD, PhD (Co-Chair)
Leigh Hochberg, MD, PhD (Co-Chair)
Session will review recent progress, with leaders in the field sharing their vision for the clinical applications and future of these important technologies
(CME Accredited Session)
Julie Parsons, MD (Chair)
Session will discuss FDA approved therapies for SMA, Duchenne Muscular Dystrophy, ALS, and Myasthenia Gravis. Strategies for medical decision making and treatment selection will be reviewed
Eric Olson, PhD (Chair)
Session will focus on recent advances and future challenges in translating CRISPR gene editing for genetic myopathies from animal models toward human translation.
Diana Bharucha-Goebel MD, MD (Chair)
Session will explore issues common to trials in rare disease such as phenotypic pleiotropy, genetic heterogeneity, combination therapies and n=1 trials.
(CME Accredited Session)
(CME Accredited Session)
Four clinicians will provide their perspective including costs, reimbursement, obtaining assistance and grant funding, hurdles, research, the role of telemedicine, altruism, and future recommendations.
We are thrilled to have had an overwhelming number of incredible abstract submissions as we reconvene in 2022. To that end, we have redesigned our Clinical Trail Presentations to include a wider range of topics which also allows for event more live presentations selected directly from the abstract submissions. This is a final day not to be missed!
