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AAN 2024 | Categorizing ALS to improve trial outcomes

The multifactorial nature of amyotrophic lateral sclerosis (ALS) results in different cohorts of patients presenting with different pathophysiology. Jinsy Andrews, MD, FAAN, from Columbia University, New York, NY, highlights the importance of considering this, as clinical trials researching experimental therapies by grouping these patients together have been largely unsuccessful. She explains the requirement for subgroups of patients to be created based on characteristic mechanisms. One such mechanism that has provoked ongoing research is gene mutations, such as in SOD1, FUS, and C9orf72 which are being targeted by antisense oligonucleotides and AAV-mediated therapies. An example of this is a recent program funded through the NIH which is identifying ultra-rare gene mutations and developing precision antisense oligonucleotides for these populations. This interview took place at the American Academy of Neurology (AAN) Annual Meeting 2024 in Denver, CO.

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