Description
+ Include: 38 videos, size: 23.9 GB
+ Target Audience: musculoskeletal physician, internal medicine physican
+ Sample video: contact me for sample video
+ Information:
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.
+ Topics:
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.mp4
ALS Collaborations A Continuum from Research to Care.mp4
ALS Track Gene directed therapy in Sporadic ALS -The story of Stathmin and More Topic.mp4
Broad range of Approaches and Study Types.mp4
Building an Infrastructure for Safe and Efficient Gene Therapy Administration.mp4
Clinical Trial Updates.mp4
Digestive Health Impacts from Top to Bottom.mp4
Digital Outcomes & Big Data Track Lab-to-Life Repairing Genetic Forms of CMT with CRISPR and More Topic.mp4
DMD 21st century innovative tools to solve a 19th century observation and More Topic.mp4
Future of Gene Therapy in NMD Considerations for Newborn Screening & Clinical Trial Design.mp4
Holistic Approach to Transitioning into Adulthood.mp4
Improving Diagnostics and Variant Interpretation.mp4
Industry Forum Assessing the Assessments for Duchenne.mp4
Industry Forum Exploring the Treatment Galaxy AAV-based Gene Transfer Research for Patients Living with Neuromuscular Diseases.mp4
Industry Forum Integrating corticosteroids into all-around Duchenne muscular dystrophy care.mp4
Industry Forum Management of Duchenne Muscular Dystrophy (DMD) How Far Have We Come.mp4
Industry Forum Targeting Fast Muscle Myosin A Novel Approach to Protecting Muscle in the Dystrophinopathies.mp4
Industry Forum Viltepso An FDA-approved Treatment for Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping.mp4
Industry Updates.mp4
Insights in Research Investor Summit.mp4
Insights into Neuromuscular Disease Mechanisms.mp4
Lab to Life Myositis.mp4
Large-Scale Data Approaches to NMD Research.mp4
Myopathies Congenital Muscular Dystrophy (CMD).mp4
NMD Care Delivery Care Throughout the Patient Journey Therapy Interventions and More Topic.mp4
NMD Care Delivery End of Life in Neuromuscular Disease Care and Considerations and More Topic.mp4
NMD Care Delivery MORV Registry Engagement Success and More Topic.mp4
NMD Care Delivery Supporting the MDA Patient Throughout Their Journey and More Topic.mp4
NMD Care Delivery The Role of Biopsy Pathology in the Era of Neuromuscular Disease Genetic Testing and More Topic.mp4
Non-Viral Delivery Approaches for Genetic Therapies.mp4
Novel Therapeutic Strategies.mp4
Opening and Keynote Address.mp4
Patient Registries and Databases.mp4
Practical Considerations in Gene Therapy Track Case Discussion.mp4
Practical Considerations in Gene Therapy Track Current and Emerging Gene Therapies for Duchenne Muscular Dystrophy and More Topic.mp4
Progress and Opportunities in ALS Therapies.mp4
Translation of Gene Editing Technologies.mp4
Ultra-Rare Track Introduction to Clinical Trials for Mitochondrial Myopathies and More Topic.mp4
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