Danon Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032
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.
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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%.
Thelansis’s “Danon Disease 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 Danon Disease treatment modalities options
for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and
China).
KOLs insights
of Danon Disease 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.
Danon Disease 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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