Danon Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2023 To 2033

Danon Disease Market Outlook

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

Danon Disease Overview

Danon Disease is a rare genetic disorder primarily affecting the skeletal and cardiac muscles, with multisystem clinical manifestations. The underlying genetic cause of the disease is a mutation in the lysosome-associated membrane protein 2 (LAMP2) gene, which leads to reduced expression of the LAMP2 protein. The reduction in LAMP2 protein expression disrupts intracytoplasmic trafficking, resulting in autophagic material and glycogen accumulation in skeletal and cardiac muscle cells. The classic presentation of DD is a triad of symptoms, including cardiomyopathy, skeletal myopathy, and intellectual disability in males. However, less prevalent symptoms, including retinal, hepatic, and pulmonary disease, may also be present. Due to the X-linked dominant inheritance pattern, male patients with LAMP2 mutations are typically affected more severely and at an earlier age than female patients. The major clinical features that suggest and confirm a diagnosis of DD include X-linked dominant inheritance, hypertrophic cardiomyopathy in young males, muscle weakness, and some degree of cognitive difficulties.

• The prevalence of Danon Disease is not well established due to limited available data. However, among individuals diagnosed with hypertrophic cardiomyopathy (approximately 1:500 individuals), the reported prevalence of those with a pathogenic LAMP2 variant has been estimated to range from 1% to 4%.

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: 2023-2033

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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