Progress & Possibilities for DM Research - 2025 MDF Conference
Автор: Myotonic Dystrophy Foundation
Загружено: 2025-08-28
Просмотров: 69
This session explores gene discoveries, gain of function RNA, and the personalized DM therapies of tomorrow, the power of unified registries, and interventional strategies targeting the cause of DM.
Presenters:
Maurice S. Swanson, PhD, Professor and Associate Director, Department of Molecular Genetics and Microbiology Center for NeuroGenetics and the Genetics Institute, University of Florida
Guillaume Bassez, MD, PhD, Institut de Myologie, MDF Medical and Scientific Advisory Committee
Antoine Mangin, PhD, Cardiff University, UK Dementia Research Institute
Explore our other sessions and read about the incredible 2025 MDF Conference in Indianapolis, Indiana: https://www.myotonic.org/over-700-peo...
Learn more about myotonic dystrophy and the Myotonic Dystrophy Foundation (MDF) at https://www.myotonic.org/
Chapters:
0:00:00 - Charting the Past: Therapeutic Strategiess for DM, Maurice S. Swanson, PhD
0:31:10 - Q&A on Therapeutic Strategiess for DM
0:35:16 - Harnessing Patient Data: Current Therapeutic Approaches and Regulatory Insights from the French Registry, Guillaume Bassez, MD, PhD
01:03:05 - Gene Correction Approach to Treat Expanded Repeat Disorders, Antoine Mangin, PhD
01:19:00 - Q&A on Gene Correction Approach to Treat Expanded Repeat Disorders
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