Niemann-Pick Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032


 Niemann-Pick disease (NPD) is a lysosomal storage disorder caused by acid sphingomyelinase deficiency (ASMD), which is characterized by the impairment of the enzyme responsible for the hydrolysis of sphingomyelin (SM) to ceramide and phosphocholine. The accumulation of SM and its precursor lipids within lysosomes, primarily in macrophages, leads to lipid-laden macrophages that deposit in various organs, including the liver, spleen, lungs, and brain. This leads to clinical symptoms such as hepatosplenomegaly, cytopenias, lung disease, and neurologic symptoms. NPD is traditionally classified into four subtypes: type A, B, C, and E. The genetic basis of NPD is inherited in an autosomal recessive pattern, meaning that both copies of the gene must have mutations for the disease to manifest. NPD types A and B are caused by missense mutations in the sphingomyelin phosphodiesterase 1 (SMPD1) gene, of which over 180 have been identified. NPD type C is caused by mutations in the NPC1 (located on chromosome 18) and NPC2 (located on chromosome 14) genes, which lead to abnormal or defective protein formation and impaired lipid transport out of cells, resulting in their accumulation within the cells. Differential diagnoses for NPD include other lysosomal storage disorders such as Gaucher disease, Tay-Sachs disease, Metachromatic leukodystrophy, and other diseases that affect the liver and brain. NPD is a progressive disorder; over time, complications can develop, such as fulminant hepatic failure, respiratory insufficiency, progressive neurodegeneration, severe thrombocytopenia, coronary artery and valvular heart disease, and deformation of bones.

·       Niemann-Pick disease type A and B affects 1 in 250,000 individuals. NPD type C affects 1 in 150,000 individuals.

Thelansis’s “Niemann-Pick Disease Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" 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 Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

KOLs insights of Niemann-Pick Disease 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 Disease 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 Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Comments

Post a Comment

Popular posts from this blog

Acne Vulgaris – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Image
  Acne vulgaris, a prevalent pilosebaceous unit inflammatory skin disorder, follows a persistent course. Typically presenting as papules, pustules, or nodules, it primarily occurs on the face but can also impact the upper arms, trunk, and back. The development of acne results from the sebaceous glands' heightened sensitivity to normal androgen levels, aggravated by Cutibacterium acnes (formerly known as Propionibacterium acnes), a bacterial species that triggers inflammation. The pathogenesis of acne vulgaris involves a complex interplay of factors such as the activation of sebaceous glands by androgens, imbalances in the pilosebaceous follicle microbiome, and cellular immune responses. Genetic predisposition and dietary influences also contribute to the condition's onset and progression. The initial stage is the micro-comedo, a small plug primarily composed of corneocytes in the lower follicular infundibulum. Microcomedones evolve into various acne lesions, including closed co...
Read more

Herpes Simplex Virus (HSV) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
 Herpes simplex virus (HSV) causes an infection that affects the external genitalia, anal region, mucosal surfaces, and skin in other body parts. Herpes simplex virus is a ubiquitous, host-adapted pathogen that causes various disease states. There are two types of HSV with two distinct DNA viruses, type 1 (HSV-1) and type 2 (HSV-2). Both the types are closely related but differ epidemiologically. HSV-1 is traditionally associated with orofacial disease by affecting the mouth and lips with fever blisters and cold sores, whereas HSV-2 is associated traditionally with genital disease infecting genitals and rectum area with sores and is classified as STD (sexually transmitted disease). As HSV-1 is associated with genital infections more often than HSV-2, lesion location is not necessarily of viral type. HSV can spread through direct contact. Almost 80% of Herpes simplex infections are asymptomatic, while symptomatic infections can be characterized by significant morbidity and recurrenc...
Read more

HR+/HER2- Breast Cancer – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Image
Thelansis’s “HR+/HER2- Breast Cancer 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 HR+/HER2- Breast Cancer treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China). KOLs insights of HR+/HER2- Breast Cancer 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. HR+/HER2- Breast Cancer 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,...
Read more