Jinsy Andrews, MD, FAAN, Columbia University, New York, NY, gives an overview of treatments for amyotrophic lateral sclerosis (ALS) that have gained approval in the last few years. She mentions two therapies that were FDA-approved in 2022: edaravone and AMX0035 (sodium phenylbutyrate/taurursodiol). Additionally, tofersen, the first gene therapy for SOD1-ALS, gained approval in 2023. However, following unsuccessful results from the confirmatory Phase III trial (NCT05021536), AMX0035 is set to be withdrawn from the market. Dr Andrews highlights that, although this withdrawal is disappointing, it shows that the regulatory process works. This interview took place at the American Academy of Neurology (AAN) Annual Meeting 2024 in Denver, CO.
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