Morquio Syndrome (Mucopolysaccharidosis Type IV) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Morquio Syndrome (Mucopolysaccharidosis Type IV) Market Outlook

Thelansis’s “Morquio Syndrome (Mucopolysaccharidosis Type IV) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 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 Morquio Syndrome (Mucopolysaccharidosis Type IV) treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Morquio Syndrome (Mucopolysaccharidosis Type IV) Overview

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.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2022-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets 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

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- 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

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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