Muscular Dystrophy Association Clinical & Scientific Conference 2023
Format: 38 videos, size: 23.9 GB
Course Audience: musculoskeletal physician, internal medicine physican
Overview:
NEW YORK, Sept. 12, 2022 /PRNewswire/ — The Muscular Dystrophy Association (MDA) today announced that registration is open for the 2023 MDA Clinical & Scientific Conference being held March 19-22, 2023. MDA is the largest worldwide convener of the neuromuscular disease community of renowned researchers, clinicians, academicians, advocates, and industry leaders both in person at the Hilton Anatole in Dallas, Texas and via live stream.
Donald S. Wood, PhD, President and CEO of the Muscular Dystrophy Association to welcome the neuromuscular disease community from around the world in March 2023.
The 2023 MDA Clinical & Scientific Conference will explore the latest research advancements and clinical achievement in neuromuscular disease with concurrent clinical and scientific sessions on topics highlighting:
- Advances in amyotrophic lateral sclerosis (ALS), Charcot-Marie-Tooth (CMT), congenital muscular dystrophy (CMD), Duchenne muscular dystrophy (DMD), Limb-girdle muscular dystrophy (LGMD), myotonic dystrophy (DM), myasthenia gravis (MG), Pompe disease, spinal muscular atrophy (SMA), and many others
- Practical considerations in delivering gene therapy
- Advances in ultra-rare neuromuscular disease
- Support for diverse clinical trial participation and clinical management
- Mechanisms in repeat-mediated diseases
- Technological advances for therapy development
- Big data – digital and computational tools (MOVR data hub)
- Clinical trial updates
“MDA’s 2023 Conference brings together global leaders in our field for exciting presentations and announcements, exploring all aspects of pre-clinical, translational, and clinical research and care across neuromuscular disease to support the development of breakthrough treatments and stronger futures for our community,” says Donald S. Wood, PhD, President & CEO, at MDA. “MDA has made a history of doing the impossible – creating the field of neuromuscular disease medicine, spearheading efforts to discover the first gene in a human disease without knowledge of that gene’s protein product, and now opening the frontiers of genetic medicine with some of the first treatments for genetic diseases in the history of medicine. What is equally important is that we convene MDA’s Care Center Network of neuromuscular disease specialists, connecting them to the latest clinical trials and FDA approved treatments. They are also developing new treatments and creating the pipeline of progress that is the hallmark of today’s neuromuscular disease frontier.”
“This year’s conference will take a particular look at emerging technologies in genetic medicine. Since the discovery of how CRISPR technology can be applied to edit genes there has been a resurgence in interest in ways that this technology can be evolved and adapted to treat neuromuscular disease,” said Sharon Hesterlee, PhD, Chief Research Officer at MDA. “In addition, new gene therapy viral vectors, non-viral gene therapy vectors and the next generation of exon-skipping drugs are all progressing rapidly. The conference will feature leading experts pioneering these new techniques.”
Before the conference, MDA will once again host the annual gathering of the Neuromuscular Advocacy Collaborative (NMAC). This pre-conference meeting brings together leaders from multiple neuromuscular advocacy organizations with the goal of developing a shared public policy agenda that will improve the lives of the entire neuromuscular disease community.
A combined stem cell and gene therapy approach to treat ALS and Stathmin-2 an emerging therapeutic target in TDP-43 proteinopathies and More Topic (Video MP4 Format)
ALS Collaborations A Continuum from Research to Care (Video MP4 Format)ALS Track Gene directed therapy in Sporadic ALS -The story of Stathmin and More Topic (Video MP4 Format)Broad range of Approaches and Study Types (Video MP4 Format)Building an Infrastructure for Safe and Efficient Gene Therapy Administration (Video MP4 Format)Clinical Trial Updates (Video MP4 Format)Digestive Health Impacts from Top to Bottom (Video MP4 Format)Digital Outcomes & Big Data Track Lab-to-Life Repairing Genetic Forms of CMT with CRISPR and More Topic (Video MP4 Format)DMD 21st century innovative tools to solve a 19th century observation and More Topic (Video MP4 Format)Future of Gene Therapy in NMD Considerations for Newborn Screening & Clinical Trial Design (Video MP4 Format)Holistic Approach to Transitioning into Adulthood (Video MP4 Format)Improving Diagnostics and Variant Interpretation (Video MP4 Format)Industry Forum Assessing the Assessments for Duchenne (Video MP4 Format)Industry Forum Exploring the Treatment Galaxy AAV-based Gene Transfer Research for Patients Living with Neuromuscular Diseases (Video MP4 Format)Industry Forum Integrating corticosteroids into all-around Duchenne muscular dystrophy care (Video MP4 Format)Industry Forum Management of Duchenne Muscular Dystrophy (DMD) How Far Have We Come (Video MP4 Format)Industry Forum Targeting Fast Muscle Myosin A Novel Approach to Protecting Muscle in the Dystrophinopathies (Video MP4 Format)Industry Forum Viltepso An FDA-approved Treatment for Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping (Video MP4 Format)Industry Updates (Video MP4 Format)Insights in Research Investor Summit (Video MP4 Format)Insights into Neuromuscular Disease Mechanisms (Video MP4 Format)Lab to Life Myositis (Video MP4 Format)Large-Scale Data Approaches to NMD Research (Video MP4 Format)Myopathies Congenital Muscular Dystrophy (CMD) (Video MP4 Format)NMD Care Delivery Care Throughout the Patient Journey Therapy Interventions and More Topic (Video MP4 Format)NMD Care Delivery End of Life in Neuromuscular Disease Care and Considerations and More Topic (Video MP4 Format)NMD Care Delivery MORV Registry Engagement Success and More Topic (Video MP4 Format)NMD Care Delivery Supporting the MDA Patient Throughout Their Journey and More Topic (Video MP4 Format)NMD Care Delivery The Role of Biopsy Pathology in the Era of Neuromuscular Disease Genetic Testing and More Topic (Video MP4 Format)Non-Viral Delivery Approaches for Genetic Therapies (Video MP4 Format)Novel Therapeutic Strategies (Video MP4 Format)Opening and Keynote Address (Video MP4 Format)Patient Registries and Databases (Video MP4 Format)Practical Considerations in Gene Therapy Track Case Discussion (Video MP4 Format)Practical Considerations in Gene Therapy Track Current and Emerging Gene Therapies for Duchenne Muscular Dystrophy and More Topic (Video MP4 Format)Progress and Opportunities in ALS Therapies (Video MP4 Format)Translation of Gene Editing Technologies (Video MP4 Format)Ultra-Rare Track Introduction to Clinical Trials for Mitochondrial Myopathies and More Topic (Video MP4 Format)