Pompe Disease – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report

Pompe disease, also known as glycogen storage disease type II (GSD2), is a metabolic disorder caused by mutations in the GAA gene on chromosome 17q25.3. These mutations lead to the synthesis of an insufficient or absent form of the lysosomal enzyme alpha-glucosidase. This results in the accumulation of glycogen within lysosomes in muscular tissue, leading to impairments in striated muscular cells. GSD2 can present as either an early-onset (infantile, classic) or late-onset (non-classic) form. Early-onset GSD2 is severe and often fatal without prompt treatment, with respiratory insufficiency and left ventricular outflow obstruction as common causes of mortality. Late-onset GSD2 also results in respiratory insufficiency but at a later age. Enzyme replacement therapy (ERT) is currently the most effective treatment for GSD2. By introducing an analogous enzyme, lysosomal glycogen accumulation in cardiac and skeletal muscle is reduced. The timing of diagnosis is a critical factor in determining the effectiveness of treatment. Differential diagnoses for early-onset GSD2 include spinal muscular atrophy 1 and Danon disease, while limb-girdle muscular dystrophy and Duchenne-Becker muscular dystrophy are differentials for late-onset.
• The estimated incidence of infantile forms of Pompe disease ranges between 1.5 to 2.7 cases per every 120,000 births, and the late-onset form ranges between 1 to 2 cases per 60,000 births.

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

KOLs insights of Pompe 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.
Pompe 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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