Amyotrophic lateral sclerosis is a rapidly progressive neurodegenerative disease affecting upper and lower motor neurons, with a median survival of two to five years from symptom onset. The majority of cases are sporadic; familial ALS accounts for roughly 10% and includes well-characterised mutations in SOD1, FUS, and C9orf72. Several RNA-targeted and small-molecule therapies have reached late-stage trials, representing the most active therapeutic pipeline in the disease's history.
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