DelveInsight Business Research LLP

DelveInsight’s “Pompe Disease Market Insights, Epidemiology, and Market Forecast-2032” report delivers an in-depth understanding of the Pompe Disease, historical and forecasted epidemiology as well as the Pompe Disease market trends in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom) and Japan 

The Pompe Disease market report provides current treatment practices, emerging drugs, market share of individual therapies, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Pompe Disease Market

• Key Companies working in the Pompe market include Genzyme, Sanofi, Amicus Therapeutics, and many others 

• Key Therapies included in the Pompe market includes Alglucosidase Alpha, Actus -101, AT845 and many others 

Pompe Disease Overview

Pompe disease, a severe metabolic myopathy, is caused by mutations in the gene coding for acid alpha-glucosidase (GAA), the enzyme that breaks down glycogen in the acidic milieu of the lysosome. Once in the lysosome, glycogen can escape following complete degradation by GAA in the form of glucose. A deficiency of the enzyme leads to lysosomal accumulation of glycogen in multiple tissues, but cardiac and skeletal muscles are most severely affected. The disease also goes by the name “Type II glycogen storage disease (GSDII)” or “Acid maltase deficiency.” It is named after a Dutch pathologist, Johannes Cassianus Pompe, who described an autopsy of a 7-month-old girl diagnosed with “idiopathic myocardial hypertrophy” and generalized muscle weakness.

Pompe Disease Epidemiology 

Pompe Disease in Europe 

• Finland has a population of approximately 5 million and has the lowest incidence of GAA deficiency, with only 2 reported cases of infantile Pompe disease and 1 case of adult-onset.3 The incidence in the Netherlands was estimated to be 1 in 40,000. Newborns were screened using newborn Guthrie cards and analyzed for the 3 most common mutations (IVS1(-13T→G), 525delT, and delexon18) seen in GAA deficiency in the Dutch population. Other mutations that account for milder disease and presentation in adulthood were not considered, so the true incidence is likely higher.

Pompe Disease in North America 

• In the United States, one study found the incidence of GAA deficiency disease to be higher than previously published estimates. The prevalence rate observed was 1 in 21,979. 

• In Mexico, a study was conducted and detected an incidence rate of 1 in 27,800.

The Report Covers the Pompe Disease Epidemiology Segmented by –

• Pompe Disease prevalent cases 

• Pompe Disease incident cases 

• Pompe Disease diagnosed cases 

• Pompe Disease treatment cases 

Click here to learn more about the Pompe Disease Market Landscape 

Pompe Disease Market Outlook 

The Pompe Disease market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Pompe Disease market trends by analyzing the impact of current Pompe Disease therapies on the market, unmet needs, drivers and barriers, and demand for better technology.

This segment gives a thorough detail of the Pompe Disease market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. According to DelveInsight, the Pompe Disease market in 7MM is expected to witness a major change in the study period 2019-2032.

Key Companies Working in the Pompe Disease Market include

• Genzyme 

• Sanofi 

• Amicus Therapeutics 

• Actus Therapeutics 

• Valerion Therapeutics

• Astellas Therapeutics 

And many others 

Pompe Disease Therapies Covered in the Report:

• Alglucosidase Alpha

• Actus -101 

• AT845

• BMN 701 

• Avalglucosidase Alfa 

• Duvoglustat

And many others 

Learn more about Key Companies and Emerging Therapies in the Pompe Disease Market 

Table of Contents

1. Key Insights 

2. Report Introduction 

3. Executive Summary of Pompe Disease Market 

4. Pompe Disease Background and Overview

5. Pompe Disease Epidemiology and patient population

6. Pompe Disease Market Emerging Therapies

7. Pompe Disease Market Cancer Market Outlook

8. Market Access and Reimbursement of Therapies

9. Appendix

10. Market Report Methodology

11. DelveInsight Capabilities

12. Disclaimer

13. About DelveInsight

Learn more about detailed report offerings @ Pompe Disease Market Outlook

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