Hurler Syndrome (Mucopolysaccharidosis Type I) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Hurler Syndrome (Mucopolysaccharidosis Type I) Market Outlook

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

Hurler Syndrome (Mucopolysaccharidosis Type I) Overview

Mucopolysaccharidosis type I (MPS I), or Hurler Syndrome, is a genetic condition that impacts various body parts. It is caused by alterations in the IDUA gene, resulting in a deficiency or complete absence of the IDUA enzyme. In the absence of this enzyme, large sugar molecules, known as glycosaminoglycans (GAGs), accumulate within cellular structures called lysosomes. This accumulation causes lysosomes to increase, leading to the enlargement of multiple organs and tissues in the body, which is responsible for the observed medical issues associated with the condition. Typically, the clinical onset of MPS I occurs between three and ten. The severity and progression of the disease can vary widely, ranging from severe, life-threatening complications that may lead to death in the second or third decade of life to a relatively longer but compromised lifespan characterized by significant disability resulting from progressive joint problems and cardiorespiratory issues. One available treatment option is hematopoietic stem cell transplantation (HSCT). This procedure can enhance cognitive function, increase survival rates, promote growth, reduce facial coarseness and hepatosplenomegaly (enlargement of the liver and spleen), improve hearing, prevent hydrocephalus (excess fluid in the brain), and alter the natural course of cardiac and respiratory symptoms.

• The incidence of Mucopolysaccharidosis type I (MPS I) ranges from 1 to 1.5 cases per 100,000 live births for the severe type and an incidence of about 1 in the 500,000 live births for the attenuated type.

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: 2021-2032

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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