Menkes Disease (MD) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Menkes Disease (MD) Market Outlook

Thelansis’s “Menkes Disease (MD) 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 Menkes Disease treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Menkes Disease (MD) Overview

Menkes disease (MD) is a rare and grave multisystemic disorder characterized by disruptions in copper metabolism. This condition, which primarily affects males due to its X-linked recessive inheritance pattern, is associated with progressive neurodegeneration, connective tissue abnormalities, and distinctive 'kinky' hair. The underlying cause of MD lies in mutations within the ATP7A gene, with most of these mutations manifesting as intragenic alterations or partial gene deletions. The ATP7A gene plays a crucial role as an energy-dependent transmembrane protein responsible for transporting copper to secreted copper enzymes while expelling excess copper from cells. This intricate process ensures the delicate balance of copper within the body, and any mutations in ATP7A can lead to the severe and life-threatening consequences observed in Menkes disease. MD primarily affects males, with most patients succumbing to the disease before reaching their third year of life. The multisystemic impact of MD includes progressive neurodegeneration, resulting in severe neurological symptoms. Additionally, connective tissue disturbances contribute to the clinical manifestations of the disease. The peculiar 'kinky' hair is a distinctive external marker of Menkes disease.

• Incidence estimates for Menkes disease in the USA range from 0.75 to 1.27 cases per 100,000 live births, highlighting its rarity. There is currently no cure for Menke's disease. However, early intervention with copper-histidine treatment, administered very early during the disease, may relieve neurological symptoms.

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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