OPENING SYMPOSIUM: The Dawn of Gene Therapy in ALS*
JEC
Time: 5:45 PM to 7:15 PM
Description
This program reviews the significant progress in our understanding of the mechanisms of action of the major mutations causing ALS in recent years, as well as the substantial progress in translational research and human therapeutic trials for these disorders, which produced the first FDA approved therapy for hereditary ALS (SOD1) in 2023.
Objectives
Describe the genetic basis of hereditary ALS.
Demonstrate how to use newly approved genetic therapies for hereditary ALS.
Explain the current landscape and timeline for the implementation of genetic therapies to the practice of ALS Care.
Speakers
A Successful RNA-Targeted Therapy for ALS
DescriptionThis presentation will describe the development of an ASO therapeutic for SOD1 ALS.
C9orf72 ALS/FTD: Challenges and New Opportunities in Translational Research
DescriptionThis session will discuss the molecular mechanisms of C9orf72 ALS/FTD, lessons from previous drug trials and new opportunities and approaches for therapeutic development.
SpeakersHuman Genetic Therapies for ALS: Current Status and Future Prospects
DescriptionUpdate on status of gene therapies in ALS including FDA approved treatment (qalsody) and new approaches for SOD1 ALS and other forms of hereditary ALS.
TDP43: A Focal Point for RNA Therapeutics in ALS/FTD
DescriptionThis session will review the connection between TDP43 and ALS, new advances that enable recognition of TDP43 pathology ante mortem, and TDP43-related therapeutics in ALS.
Speakers