Mucopolysaccharidosis Type IIIA (MPS IIIA) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033 thelansis.com

Mucopolysaccharidosis type III (MPS III), part of the mucopolysaccharidoses group, is a lysosomal storage disease characterized by severe and rapid intellectual deterioration. Initial symptoms typically manifest between the ages of 2 and 6 years, presenting with behavioral disorders such as hyperkinesia and aggressiveness, along with intellectual decline, sleep disturbances, and mild dysmorphism. Neurological involvement becomes more pronounced around age 10, leading to losing motor milestones and communication difficulties. Seizures often occur after age 10; some attenuated forms have been reported. Each MPS III subtype (MPS IIIA, MPS IIIB, MPS IIIC, and MPS IIID) results from deficiencies in one of the four enzymes necessary for heparan sulfate (HS) degradation. Specific enzymes responsible for each subtype include heparan sulfamidase for MPS IIIA, alpha-N-acetylglucosaminidase for MPS IIIB, alpha-glucosaminide N-acetyltransferase for MPS IIIC, and N-acetylglucosamine-6-sulfate sulfatase for MPS IIID. The respective genes for these enzymes have been identified (MPS IIIA on 17q25, MPS IIIB on 17q21, MPS IIIC in the pericentromeric region of chromosome 8, MPS IIID on 12q14), with numerous associated mutations. Allogenic bone marrow grafts are contraindicated, as they do not impede mental deterioration, even in pre-symptomatically engrafted patients. Gene therapy is currently being investigated in animal models for MPS IIIA and MPS IIIB subtypes. The complex neurological degradation and multiple complications necessitate a multidisciplinary approach to provide tailored symptomatic treatment.
• The incidence of MPS in the US has been reported as 0.98 per 100,000 live births.

Thelansis’s “Mucopolysaccharidosis Type IIIA (MPS IIIA) 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 Mucopolysaccharidosis Type IIIA (MPS IIIA) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

KOLs insights of Mucopolysaccharidosis Type IIIA (MPS IIIA) 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.

Mucopolysaccharidosis Type IIIA (MPS IIIA) 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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