Morquio Syndrome (Mucopolysaccharidosis Type IV) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033


 

Morquio Syndrome, also known as Mucopolysaccharidosis Type IV, is a rare lysosomal storage disease with mild to severe spondylosis-epiphyses-metaphyseal dysplasia. It is characterized by disproportionate short stature with a short neck and trunk, joint laxity, pectus carinatum, genum valgum, abnormal gait, tracheal narrowing, spinal abnormalities such as kyphosis and scoliosis, respiratory impairment, and valvular heart disease. Clinically, the two forms of MPS, IVA and IVB, have similar skeletal manifestations. But MPS IVA is more severe. MPS IVA is diagnosed during the second year of life. Progressive skeletal and joint deformities lead to impairment in walking and daily activities. Patients experience low endurance, debilitating fatigue, and pain; many become wheelchair-dependent in their second decade. Body-wide accumulation of glycosaminoglycans (GAGs) results in dysfunction and dysplasia. In MPS IVA, deficiency of the enzyme N-acetylgalactosamine-6-sulfate sulfatase (GALNS, 16q24.3) required for keratin sulfate (KS) degradation and chondroitin 6-sulfate leads to GAG accumulation. MPS IVB is caused by a deficiency in beta-galactosidase (GLB1, 3p22.3) required for KS degradation. Without treatment, MPS IVA patients generally do not survive beyond their third decade, but with appropriate management, patients can live beyond age 50, with some surviving over 70 years old.

  • The estimated incidence of Mucopolysaccharidosis Type IV in the USA is 0.14 cases per 100,000.

 

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

KOLs insights of Morquio Syndrome (Mucopolysaccharidosis Type IV) 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.

Morquio Syndrome (Mucopolysaccharidosis Type IV) 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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