Thelansis

 Niemann-Pick Type C (NPC) is a group of disorders that involve abnormalities of intracellular transport of endocytosed cholesterol within cells, resulting in the accumulation of unesterified cholesterol in lysosomes and late endosomes. This condition is inherited in an autosomal recessive manner and caused by mutations in the NPC1 or NPC2 genes. The functions of these proteins still need to be fully understood. NPC can be classified into three subtypes based on the age of onset of neurological manifestations: 1.        Visceral-neurodegenerative form: Early-infantile (< 2 years) 2.        Neurodegenerative form: Late-infantile (2–6 years), Juvenile (6–15 years) 3.        Psychiatric-neurodegenerative form: Adult (> 15 years) Symptoms vary depending on the age of onset and may include jaundice, hepatosplenomegaly, ataxia, dysarthria, cognitive decline, seizures, dysphagia, and ca...

 Niemann-Pick Type C (NPC) is a progressive and life-limiting autosomal recessive disorder caused by mutations in the NPC1 or NPC2 gene. Mutations in these genes are associated with abnormal endosomal-lysosomal trafficking, resulting in the accumulation of multiple tissue-specific lipids in the lysosomes and abnormal accumulation of these substances within various tissues of the body, including brain tissue. ·        Niemann-Pick Type C (NPC) is estimated to have a minimal incidence of 1/120 000 live births in the US. ·        Estimates of birth prevalence ranging between 0.26 and 0.94 per 100,000 were proposed for France, the UK, and Germany, with at least 95% of all diseases due to mutations in the NPC1 gene and the remainder in the NPC2 gene. Thelansis’s “Niemann-Pick Type C (NPC) Market Outlook, Epidemiology, Competitive Landscape , and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology,...