Repurposed ALS (amyotrophic lateral sclerosis) drug may help slow down a rare genetic disorder in children and adolescents.
Amyotrophic Lateral Sclerosis (ALS)-Lou Gehrig's disease: Symptoms, Diagnosis, Treatment
Find out more about the degenerative disease- Amyotrophic lateral sclerosis.
Find out more about the degenerative disease- Amyotrophic lateral sclerosis.
‘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.
Niemann-Pick Disease - Types, Causes, Symptoms, Diagnosis & Treatment
Niemann–Pick disease is a group of inherited metabolic disorders in which cholesterol and lipids accumulate in lysosomes, leading to organ damage.
Niemann–Pick disease is a group of inherited metabolic disorders in which cholesterol and lipids accumulate in lysosomes, leading to organ damage.
Direct Brain Administration Increases Efficacy of Niemann-Pick Disease Treatment
Clinical trial data suggests that direct administration to the central nervous system provides the greatest Niemann-Pick Disease neurological efficacy.
Clinical trial data suggests that direct administration to the central nervous system provides the greatest Niemann-Pick Disease neurological efficacy.
