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Repurposed Amyotrophic Lateral Sclerosis (ALS) Drug Against Rare Genetic Disorder

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Riluzole, a repurposed ALS (amyotrophic lateral sclerosis) drug may help slow down a rare genetic disorder called Niemann-Pick disease type C1 (NPC1 - characterized by the gradual loss of a particular brain cell) in children and adolescents, as per a new study in mice at the National Institutes of Health, published in i Molecular Genetics and Metabolism. /i NPC1 occurs due to an b impaired ability to move cholesterol through cells. /b This leads to difficulty in controlling movements, ...

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