As ALS patients unite around Amylyx, a new paper hints at potential druggable targets
ALS is a debilitating, universally fatal disease. As motor neurons die, patients lose their abilities to walk, cut their own food, swallow and eventually, breathe. Most patients die within three to five years of symptom onset, and there are few approved treatments that only modestly impact function and survival.
Though patients and advocates have rallied around an experimental drug from Amylyx Pharmaceuticals, currently being reviewed by the FDA, they continue to emphasize how more work needs to be done. Research into ALS pathology remains scant relative to other fields, as scientists have yet to discover a confirmed biomarker that measures patients’ progress and have only identified a handful of genetic targets implicated in the disease.
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