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20211119 als.
NPC1 leads to difficulty controlling movements, liver and lung disease, impaired swallowing, intellectual decline and death. Riluzole, a drug approved to treat amyotrophic lateral sclerosis (ALS), a disease affecting nerve cells controlling movement, could slow the gradual loss of a particular brain cell that occurs in Niemann-Pick disease type C1 (NPC1), a rare genetic disorder affecting children and adolescents, suggests ...
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