ALS is a motor neuron disease (MND™), is a debilitating, fatal, late-onset degenerative disorder of the nervous system characterized by motor neuron degeneration, which leads to progressive muscle wasting and weakness. The disease progresses rapidly; the median survival is three to five years, and only 20% of patients are alive five to ten years after symptom onset. Death usually results from respiratory failure. Despite the poor prognosis, there is considerable variation in the survival rate, and up to 10% of people with ALS™ live for more than eight years from the onset of first symptoms. The major breakthroughs in the etiology and pathophysiology of ALS™ are the discovery of various genetic risk factors such as mutations in SOD1, TARDBP, FUS, C9orf72, TBK1, and NEK1. Most of these risk factors were discovered only within the last ten years, and extensive research is underway to uncover the common mechanisms affected by these mutations that may lead to ALS, such as protein misfolding and aggregation.
Target patient pool; ALS prevalence in the United States ranges between 5.2 to 6.5 cases per 100,000 population where as incidence cases ranges between 0.6 to 2.8 cases per 100,000 population.
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