Acid Sphingomyelinase Deficiency (ASMD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032
Acid Sphingomyelinase Deficiency (ASMD), a rare lysosomal storage disease, is an autosomal recessive genetic disorder caused by mutations in the SMPD1 gene. ASMD has been classified as; Niemann-Pick disease type A (NPD A) and Niemann-Pick disease type B (NPD B). More than 180 SMPD1 mutations are identified in patients with ASMD, including missense, nonsense, frameshift, and splice variants. Diagnoses incidence of ASMD at approximately 0.5 per 100,000 births. NPD A patients have a relatively uniform natural history characterized by severe progressive neurodegeneration in the first year and death typically by three years of age.
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NPD B has a variable disease course associated
with a broad spectrum of disease severity and manifestations.
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Clinical manifestations of ASMD are
hepatosplenomegaly, developmental delay and/or cherry-red maculae, interstitial
lung disease, and hyperlipidemia characterized by low high-density lipoprotein
(HDL) cholesterol and/or thrombocytopenia.
Thelansis’s “Acid Sphingomyelinase
Deficiency (ASMD) 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 Acid
Sphingomyelinase Deficiency (ASMD) treatment modalities options for eight major
markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
KOLs insights
of Acid Sphingomyelinase Deficiency (ASMD) 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.
Acid Sphingomyelinase Deficiency
(ASMD) 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.
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