Acid Sphingomyelinase Deficiency (ASMD) – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030


 Acid sphingomyelinase (ASM) 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 different SMPD1 mutations are identified in patients with ASMD, including missense, nonsense, frameshift mutations, 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. NPD B has a variable disease course associated with a broad spectrum of disease severity and manifestations.

Clinical manifestations of ASMD are hepatosplenomegaly, developmental delay, cherry-red maculae, interstitial lung disease, hyperlipidemia characterized by low high-density lipoprotein (HDL) cholesterol, and thrombocytopenia.

 

The competitive landscape of Acid Sphingomyelinase Deficiency (ASMD) includes country-specific approved and pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are tracked and supplemented with analyst commentary.

KOLs insights of Acid Sphingomyelinase Deficiency (ASMD) across 8 MM market from the center 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.

Read more: Acid Sphingomyelinase Deficiency (ASMD) – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          olipudase alfa    Genzyme          Phase 2/3

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