Niemann-Pick Type C (NPC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033


 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 cataplexy. The differential diagnosis may consist of other lipidoses; idiopathic neonatal hepatitis and other causes of cholestatic icterus should be considered in neonates, and conditions with cerebellar ataxia, dystonia, cataplexy, and supranuclear gaze palsy in older children and adults. Apart from Zavesca in Europe, no approved therapies for managing symptoms manifest as Niemann–Pick disease.

·       At least 95% of all diseases are due to mutations in the NPC1 gene and the remainder in the NPC2 gene. NPC is estimated to have a minimal incidence of 1/120 000 live births in the US. Birth prevalence estimates between 0.26 and 0.94 per 100,000 were proposed for France, the UK, and Germany.

 

Thelansis’s “Niemann-Pick Type C (NPC) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Niemann-Pick Type C (NPC) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

 

KOLs insights of Niemann-Pick Type C (NPC) across 8 MM market from the centre of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Niemann-Pick Type C (NPC) Market Forecast Patient Based Forecast Model (MS. Excel Based Automated Dashboard), which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Thelansis Competitive Intelligence (CI) practice has been established based on a deep understanding of the pharma/biotech business environment to provide an optimized support system to all levels of the decision-making process. It enables business leaders in forward-thinking and proactive decision-making. Thelansis supports scientific and commercial teams in seamless CI support by creating an AI/ ML-based technology-driven platform that manages the data flow from primary and secondary sources.

Read more: Niemann-Pick Type C (NPC) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Tags: Niemann-Pick Type C (NPC), Niemann-Pick Type C (NPC) market outlook, Niemann-Pick Type C (NPC) competitive landscape, Niemann-Pick Type C (NPC) market forecast, Thelansis, Primary market research, KOL insights, Competitive Intelligence (CI)

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