The Pyruvate Kinase Deficiency market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Pyruvate Kinase Deficiency: An Overview
Pyruvate Kinase Deficiency (PKD) is a rare genetic disorder, that signifies the lack of the enzyme pyruvate kinase, which is used by red blood cells. Without this enzyme, red blood cells break down too easily (premature destruction), resulting in a low level of these cells (haemolytic anaemia). Anaemia is a general term for when there are low levels of red blood cells in the bloodstream, and haemolytic (or haemolysis) means that the red blood cells break down prematurely.
Mild cases require no treatment. People with severe anaemia may need blood transfusions. In newborns with dangerous levels of jaundice, a health care provider may recommend an exchange transfusion. Surgical removal of the spleen (splenectomy) may also be necessary to help reduce the destruction of red blood cells. Another treatment is symptomatic and supportive. Someone who had a splenectomy should receive the pneumococcal vaccine at recommended intervals.
Pyruvate Kinase Deficiency Market Key Facts
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Pyruvate Kinase Deficiency is a rare disorder that affects both men and women. The frequency of the disorder is unknown, although one estimate suggests that approximately 1 in 20,000 Caucasian people develop the disorder.
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Pyruvate Kinase Deficiency has a worldwide distribution, although it is more prevalent in Northern Europe, Japan, and China. The global incidence is around five cases per population of one million. In the United States, the incidence is 1 in 20 000, and most cases are identified via prenatal genetic screening. The estimated prevalence of the disease is 51 per million population. The frequency of the disease is higher among the Pennsylvania Amish and Romani communities.
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As per the National Organization for Rare Disorders, in clinical practice, the frequency is closer to 1 in 1,000,000 people. PKD has been identified most commonly in Europe. However, rare disorders like PKD often go misdiagnosed or undiagnosed making it difficult to determine their true frequency in the general population.
Pyruvate Kinase Deficiency Market
The Pyruvate Kinase Deficiency market size is expected to rise during the forecast period owing to the rise in the number of prevalent cases in 7MM.
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted Pyruvate Kinase Deficiency market size by analyzing the impact of current and emerging therapies in the market. It also provides a thorough assessment of the market drivers & barriers, unmet needs, and emerging technologies.
The report gives complete detail of the Pyruvate Kinase Deficiency market trend for each marketed drug and late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, mechanism of action (MoA), competition with other therapies, brand value, and their impact on the market.
Pyruvate Kinase Deficiency Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of diagnosed patient pool and future trends.
Pyruvate Kinase Deficiency Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Pyruvate Kinase Deficiency market or expected to get launched during the study period. The analysis covers Pyruvate Kinase Deficiency market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Pyruvate Kinase Deficiency Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Pyruvate Kinase Deficiency Therapeutics Analysis
As per DelveInsight, the Pyruvate Kinase Deficiency (PKD) market dynamics is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, incremental healthcare spending across the world, and also the expected launch of emerging therapies.
The current pipeline for Pyruvate Kinase Deficiency (PKD) does not hold many significant products, it has very few products that are being developed by certain key players.
Research into new treatments for Pyruvate Kinase Deficiency is very promising. The novel treatment approaches, which range from a small molecule pyruvate kinase activator to gene therapy, may transform the way in which PKD is managed in the future.
Some of the key companies in the Pyruvate Kinase Deficiency Market include:
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Agios Pharmaceuticals
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Rocket Pharmaceuticals
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Centogene AG, and many others.
Pyruvate Kinase Deficiency Therapies covered in the report include:
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Mitapivat
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RP-L301
And many more.
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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Pyruvate Kinase Deficiency Competitive Intelligence Analysis
4. Pyruvate Kinase Deficiency Market Overview at a Glance
5. Pyruvate Kinase Deficiency Disease Background and Overview
6. Pyruvate Kinase Deficiency Patient Journey
7. Pyruvate Kinase Deficiency Epidemiology and Patient Population
8. Pyruvate Kinase Deficiency Treatment Algorithm, Current Treatment, and Medical Practices
9. Pyruvate Kinase Deficiency Unmet Needs
10. Key Endpoints of Pyruvate Kinase Deficiency Treatment
11. Pyruvate Kinase Deficiency Marketed Products
12. Pyruvate Kinase Deficiency Emerging Therapies
13. Pyruvate Kinase Deficiency Seven Major Market Analysis
14. Attribute Analysis
15. Pyruvate Kinase Deficiency Market Outlook (7 major markets)
16. Pyruvate Kinase Deficiency Access and Reimbursement Overview
17. KOL Views on the Pyruvate Kinase Deficiency Market.
18. Pyruvate Kinase Deficiency Market Drivers
19. Pyruvate Kinase Deficiency Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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About DelveInsight
DelveInsight is a leading Business Consultant and Market Research Firm focused exclusively on life sciences. It supports pharma companies by providing end-to-end comprehensive solutions to improve their performance.
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