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The Pyruvate Kinase Deficiency market report provides an edge while developing business strategies, by understanding trends shaping and driving the global Pyruvate Kinase Deficiency (PKD) market.

Pyruvate Kinase Deficiency Overview

Pyruvate Kinase Deficiency (PKD) is a rare genetic disorder, 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.

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List of regions covered in the report

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Pyruvate Kinase Deficiency Causes

PKD is caused by alterations (mutations) in the PKLR gene, which lead to a deficiency of the enzyme pyruvate kinase. These genetic alterations are inherited in an autosomal recessive manner. Pyruvate kinase is an enzyme that helps cells turn sugar (glucose) into energy (called adenosine triphosphate, ATP) in a process called glycolysis. Red cells rely on this process for energy, and so, pyruvate kinase deficiency leads to a deficiency in energy and to premature red cell destruction (haemolysis). Instead of lasting 120 days, red cells with pyruvate kinase deficiency last only a few days to weeks.

Pyruvate Kinase Deficiency Symptoms

PKD symptoms usually include the breakdown of red blood cells resulting in haemolytic anaemia, a yellowing of the whites of the eyes (icterus), fatigue, lethargy, recurrent gallstones, jaundice, and pale skin (pallor). In more severe cases, the first signs and symptoms may appear in utero in the form of hydrops fetalis, a condition in which abnormal amounts of fluid buildup in two or more body areas of the foetus. Newborns may present with prolonged jaundice and anaemia. Older children may be pale (due to anaemia) and have intermittent episodes of jaundice. Mild cases may escape detection until adulthood. Although the anaemia tends to stabilize in adulthood, episodes of anaemia may occur with acute infections, stress, and pregnancy.

Pyruvate Kinase Deficiency Market Report

Mild cases require no treatment. People with severe anemia 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. Other treatment is symptomatic and supportive. Someone who had a splenectomy should receive the pneumococcal vaccine at recommended intervals.

Pyruvate Kinase Deficiency Treatment Market

PKD treatment is predominantly supportive as the available curative measures have a very high risk that outweighs the benefit of attempting them. The most important step is to control the associated anaemia and its complications. This can be done by: Simple transfusion, Supplemental folic acid and vitamins, Chelation therapy, Phototherapy and Splenectomy.

Pyruvate Kinase Deficiency Market Insights

Pyruvate Kinase Deficiency (PKD) is an autosomal recessive inherited disease that produces ineffective conversion of phosphoenolpyruvate in the Embden-Meyerhof Pathway, resulting in decreased production of ATP. It is the second most frequent enzymatic defect after G-6-P-D deficiency.

Pyruvate Kinase Deficiency Market Report Scope

• The report covers the descriptive overview of Pyruvate Kinase Deficiency, explaining its causes, signs and symptoms, pathophysiology, diagnosis and currently available therapies
• Comprehensive insight has been provided into the Pyruvate Kinase Deficiency epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Pyruvate Kinase Deficiency are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
• A detailed review of Pyruvate Kinase Deficiency market; historical and forecasted is included in the report, covering drug outreach in the 7MM
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Pyruvate Kinase Deficiency market

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Table of content

1. Key Insights

2. Executive Summary of Pyruvate Kinase Deficiency

3. Competitive Intelligence Analysis for Pyruvate Kinase Deficiency

4. Pyruvate Kinase Deficiency: Market Overview at a Glance

4.1. Pyruvate Kinase Deficiency Total Market Share (%) Distribution in 2017

4.2. Pyruvate Kinase Deficiency Total Market Share (%) Distribution in 2030

5. Pyruvate Kinase Deficiency: Disease Background and Overview

6. Patient Journey

7. Pyruvate Kinase Deficiency Epidemiology and Patient Population

8. Treatment Algorithm, Current Treatment, and Medical Practices

9. Unmet Needs

10. Key Endpoints of Pyruvate Kinase Deficiency Treatment

11. Marketed Products

12. Emerging Therapies

13. Pyruvate Kinase Deficiency: Seven Major Market Analysis

14. Attribute analysis

15. 7MM: Market Outlook

16. Access and Reimbursement Overview of Pyruvate Kinase Deficiency

17. KOL Views

18. Market Drivers

19. Market Barriers

20. Appendix

21. DelveInsight Capabilities

22. Disclaimer

23. About DelveInsight

Pyruvate Kinase Deficiency Market Report Highlights

• In the coming years, the Pyruvate Kinase Deficiency market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
• The companies and academics are working to assess challenges and seek opportunities that could influence Pyruvate Kinase Deficiency R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition      
• Major players are involved in developing therapies for Pyruvate Kinase Deficiency. Launch of emerging therapies will significantly impact the Pyruvate Kinase Deficiency market
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Pyruvate Kinase Deficiency
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Why should you buy this report?

• The report will help in developing business strategies by understanding trends shaping and driving the Pyruvate Kinase Deficiency market
• To understand the future market competition in the Pyruvate Kinase Deficiency market and Insightful review of the key market drivers and barriers
• Organize sales and marketing efforts by identifying the best opportunities for Pyruvate Kinase Deficiency in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors
• Organize sales and marketing efforts by identifying the best opportunities for the Pyruvate Kinase Deficiency market
• To understand the future market competition in the Pyruvate Kinase Deficiency market

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