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 Molecular Genetics and Metabolism.
NPC1 occurs due to an impaired ability to move cholesterol through cells. This leads to difficulty in controlling movements, liver and lung disease, impaired swallowing, intellectual decline, and death, as a result of gradual loss of brain cells known as Purkinje neurons.
‘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.
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The present study found that on treating the NPC1 mice riluzole, there was an improved survival of 12% longer as compared to untreated mice.
This showed that riluzole or similar drugs may help slow down the disease progression in NPC1patients.
Source: Medindia
