Advances in Amyotrophic Lateral Sclerosis*

Date/Time: Sunday, October 17, 2021 - 3:15 PM – 5:15 PM
Track: Plenary Session
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Description:

The pace of discovery has accelerated, and there are glimmers of hope emerging for the 20,000 Americans living with amyotrophic lateral sclerosis (ALS). This session will outline recent advances in ALS that underlie this excitement, including new treatments, clinical trial design, large-scale initiatives, insights into the disease provided by genetics, and future directions. A series of field leaders will describe their research and what excites them moving forward. In addition to exciting advances in therapy based on small molecules, gene therapy has emerged as a disease-altering treatment in an otherwise fatal disease. We summarize the novel genetic discoveries associated with ALS, demonstrating the technologies and large-scale experimental design used to identify these genes. These genetic discoveries will be discussed in the context of developing new therapies. Innovative clinical trial designs that streamline and accelerate the testing of therapies are coming to the fore, as well as the use of remote sensing to increase patient engagement in clinical trials. In many ways, ALS acts as a critical testbed for neurodegenerative diseases in general. Therefore, both the general neurologist and those specializing in ALS will benefit from the detailed description of the topics.

Chair: Bryan Traynor, MD, PhD

Co-Chair: Sabrina Paganoni MD, PhD

Objective(s):

  • Understand the recent advances in our understanding and treatment of amyotrophic lateral sclerosis

  • Follow the recent advances in our genetic understanding of amyotrophic lateral sclerosis

  • Recognize the potential of targeted gene therapy in treating inherited forms of ALS 

  • Know how new trial designs are revolutionizing clinical trials in ALS

Antisense Oligonucleotide Therapy for SOD1 ALS

Time: 3:15 PM – 5:15 PM

Mapping of Critical Events in ALS Progression

Time: 3:15 PM – 5:15 PM

Speaker(s):

Recent Advances in ALS

Time: 3:15 PM – 5:15 PM

Regulation of TDP-43 Nucleocytoplasmic Shuttling and Therapeutic Targeting

Time: 3:15 PM – 5:15 PM

Unraveling the Genomics of ALS

Time: 3:15 PM – 5:15 PM