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Pompe Disease Market

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

The Pompe Disease market report provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Pompe Disease market size from 2019 to 2032, segmented by seven major markets. The Report also covers current Pompe Disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Pompe Disease market.

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Some facts of the Pompe Disease Market Report are:

• According to DelveInsight, Pompe Disease market size is expected to grow at a decent CAGR by 2032.
• Leading Pompe Disease companies working in the market are Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics, Novartis AG, Fresenius Kabi AG, Akorn Incorporated, Teva Pharmaceutical, Mylan N.V, Johnsons & Johnsons, F. Hoffman-La Roche Ltd., Trinity Biotech plc, and others.
• Key Pompe Disease Therapies expected to launch in the market are Cipaglucosidase alfa, SPK-3006, ACTUS 101, AT845, RP A501, AOC-Pompe disease, AVR RD 03, GYS1 Program, ETV-GAA, and many others.
• On April 2024, Astellas Gene Therapies announced results of a Study to Evaluate Seroprevalence of Antibodies to AAV8 and Assessment of Biomarkers in Patients With Late-Onset Pompe Disease.
• On April 2024, Amicus Therapeutics announced results of an Open-Label, Fixed-Sequence, Ascending-Dose, First-in-Human Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of Intravenous Infusions of ATB200 Co-Administered With Oral AT2221 in Adult Subjects With Pompe Disease.
• On March 2024, Genzyme, a Sanofi Company announced results of a Single Arm, Prospective, Open-label, Multi-center Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatment.
• On November 2023, Spark Therapeutics, Inc announced results of a Phase 1/2, Dose-escalation Study to Evaluate the Safety, Tolerability and Efficacy of a Single Intravenous Infusion of SPK-3006 in Adults With Late-onset Pompe Disease.
• On June 2023, Asklepios Biopharmaceutical, Inc announced results of a Phase 1 Study of the Safety of AAV2/8-LSPhGAA (ACTUS-101) in Late-onset Pompe Disease.

Pompe Disease Overview

Pompe disease, also known as glycogen storage disease type II, is a rare genetic disorder characterized by the buildup of glycogen in cells, particularly within muscles. This accumulation impairs the function of various organs, predominantly affecting the muscles, heart, and liver. Pompe disease is caused by mutations in the GAA gene, leading to deficiency or dysfunction of the enzyme acid alpha-glucosidase (GAA), which is responsible for breaking down glycogen. Symptoms can vary widely in severity and may include muscle weakness, respiratory difficulties, enlarged heart (cardiomegaly), and liver problems. Early onset Pompe disease typically presents in infancy, while late-onset forms may manifest later in childhood or adulthood. Treatment options include enzyme replacement therapy (ERT) to supplement the deficient enzyme and manage symptoms, along with supportive care to address specific complications. Regular monitoring and management by healthcare professionals are essential for optimizing outcomes in individuals with Pompe disease.

Learn more about Pompe Disease treatment algorithms in different geographies, and patient journeys. Contact to receive a sample @ https://www.delveinsight.com/sample-request/pompe-disease-market

Pompe Disease Market 

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 and unmet needs, and drivers, 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. The calculated Pompe Disease market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Pompe Disease market in 7MM is expected to witness a major change in the study period 2019-2032.

Pompe Disease Epidemiology

The Pompe Disease epidemiology section provides insights into the historical and current Pompe Disease patient pool and forecasted trends for seven individual major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Pompe Disease market report also provides the diagnosed patient pool, trends, and assumptions. 

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Pompe Disease Drugs Uptake

This section focuses on the uptake rate of the potential Pompe Disease drugs recently launched in the Pompe Disease market or expected to be launched in 2019-2032. The analysis covers the Pompe Disease market uptake by drugs, patient uptake by therapies, and sales of each drug.

Pompe Disease Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Pompe Disease market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Pompe Disease Pipeline Development Activities

The Pompe Disease report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Pompe Disease key players involved in developing targeted therapeutics.

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Pompe Disease Therapeutics Assessment

Major key companies are working proactively in the Pompe Disease Therapeutics market to develop novel therapies which will drive the Pompe Disease treatment markets in the upcoming years are Spark Therapeutics, Amicus Therapeutics, Asklepios Biopharmaceutical, Audentes Therapeutics, Rocket Pharmaceuticals, Avidity Biosciences, AVROBIO, Maze Therapeutics, Denali Therapeutics, Novartis AG, Fresenius Kabi AG, Akorn Incorporated, Teva Pharmaceutical, Mylan N.V, Johnsons & Johnsons, F. Hoffman-La Roche Ltd., Trinity Biotech plc, and others.

Learn more about the emerging Pompe Disease therapies & key companies @ https://www.delveinsight.com/sample-request/pompe-disease-market

Pompe Disease Report Key Insights

1. Pompe Disease Patient Population

2. Pompe Disease Market Size and Trends

3. Key Cross Competition in the Pompe Disease Market

4. Pompe Disease Market Dynamics (Key Drivers and Barriers)

5. Pompe Disease Market Opportunities

6. Pompe Disease Therapeutic Approaches

7. Pompe Disease Pipeline Analysis

8. Pompe Disease Current Treatment Practices/Algorithm

9. Impact of Emerging Therapies on the Pompe Disease Market

Table of Contents

1. Key Insights

2. Executive Summary

3. Pompe Disease Competitive Intelligence Analysis

4. Pompe Disease Market Overview at a Glance

5. Pompe Disease Disease Background and Overview

6. Pompe Disease Patient Journey

7. Pompe Disease Epidemiology and Patient Population

8. Pompe Disease Treatment Algorithm, Current Treatment, and Medical Practices

9. Pompe Disease Unmet Needs

10. Key Endpoints of Pompe Disease Treatment

11. Pompe Disease Marketed Products

12. Pompe Disease Emerging Therapies

13. Pompe Disease Seven Major Market Analysis

14. Attribute Analysis

15. Pompe Disease Market Outlook (7 major markets)

16. Pompe Disease Access and Reimbursement Overview

17. KOL Views on the Pompe Disease Market

18. Pompe Disease Market Drivers

19. Pompe Disease Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

About DelveInsight

DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.

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