ALS Finding a Cure Providing the Initial Funding for Amylyx In every journey of discovery, certain days distinguish themselves as fundamental to achieving the ultimate objective. Yesterday was one of those days in the fight against ALS, Lou Gehrig’s Disease, with the FDA’s approval of Amylyx’s new drug, Relyvrio, the first new ALS drug in over five years. ALS (amyotrophic lateral sclerosis) is a devastating disease of the nervous system which robs patients of their muscle control and eventually their ability to breathe on their own.  While rare, there is no known cure, and it famously struck down Lou [...]

Publications related to Answer ALS, a project ALS FAC helped fund 1. Baxi, E. G. et al. Answer ALS, a large-scale resource for sporadic and familial ALS combining clinical and multi-omics data from induced pluripotent cell lines. Nat Neurosci 1-12 (2022) doi:10.1038/s41593-021-01006-0. 2. Zhang, S. et al. Genome-wide identification of the genetic basis of amyotrophic lateral sclerosis. Neuron (2022) doi:10.1016/j.neuron.2021.12.019. 3. Gilley, J. et al. Enrichment of SARM1 alleles encoding variants with constitutively hyperactive NADase in patients with ALS and other motor nerve disorders. Medrxiv 2021.06.17.21258268 (2021) doi:10.1101/2021.06.17.21258268. 4. Ramamoorthy, D. et al. Identifying Patterns of ALS Progression from Sparse Longitudinal Data. Medrxiv 2021.05.13.21254848 (2021) doi:10.1101/2021.05.13.21254848. 5. Coyne, A. N. et al. Nuclear accumulation of CHMP7 [...]

A panel of independent advisers to the US Food and Drug Administration voted Wednesday (September 7) to recommend the agency approve a novel therapy to treat amyotrophic lateral sclerosis, otherwise known as Lou Gehrig’s disease. The drug, called AMX0035, had previously been rejected by the same group earlier in the year, but new evidence presented by the company that developed it, Massachusetts-based Amylyx Pharmaceuticals, prompted a surprising reversal that also comes amid an aggressive lobbying campaign by ALS patients and their families. Read More...

Scientists have identified several potential therapeutic targets for amyotrophic lateral sclerosis (ALS) using an artificial intelligence (AI)-powered target discovery engine to analyze dysregulated gene expression in large, publicly available ALS datasets.

Twelve-month data show that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength and quality of life Tofersen also led to robust and sustained reductions in neurofilament, a marker of neurodegeneration SOD1-ALS is a rare, progressive and fatal genetic form of the disease leading to loss of everyday functions and affecting approximately 2% of people with ALS

IRVINE, Calif., May 31, 2022 (GLOBE NEWSWIRE) -- Eledon Pharmaceuticals, Inc. (“Eledon”) (Nasdaq: ELDN), a patient-focused clinical stage biopharmaceutical company committed to the development of innovative and impactful treatments for organ and cell transplantation, autoimmune conditions, and neurodegenerative disease, today announced topline results from a Phase 2a clinical trial of tegoprubart (formerly AT-1501) in patients with amyotrophic lateral sclerosis (ALS).1