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.
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.
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The Pyruvate Kinase Deficiency market report also covers emerging drugs, current treatment practices, Pyruvate Kinase Deficiency market share of the individual therapies, current and forecasted Pyruvate Kinase Deficiency Market Size from 2017 to 2030 segmented by seven major markets.
The report provides a detailed current Pyruvate Kinase Deficiency treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
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.
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The various research findings on Pyruvate Kinase Deficiency (PKD) concluded that: PKD is rare, estimated to occur in three to eight per 1,000,000 people. It is equally common in men and women. [Boston Children’s Hospital]
Key Benefits of Pyruvate Kinase Deficiency Market Report
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Pyruvate Kinase Deficiency market report provides an in-depth analysis of Pyruvate Kinase Deficiency Market Size, Share, and Trend in 7 major markets, i.e. EU5 (Germany, Italy, Spain, France, and the UK), Japan, and the United States.
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The Pyruvate Kinase Deficiency market report will help in developing business strategies by understanding the Pyruvate Kinase Deficiency Market trends & developments, key players, and future market competition that will shape and drive the Pyruvate Kinase Deficiency market in the upcoming years.
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The Pyruvate Kinase Deficiency market report covers Pyruvate Kinase Deficiency current treatment practices, emerging drugs, market share of the individual therapies in 7 MM.
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The report provides a detailed assessment of the Pyruvate Kinase Deficiency market in terms of market drivers & barriers, Unmet Needs, market opportunities, patient population, comparative analysis of pipeline products with detailed clinical profiles, and other factors.
Pyruvate Kinase Deficiency Market
Pyruvate Kinase Deficiency market size is expected to rise during the forecast period owing to the rise in the number of Prevalent cases of Pyruvate Kinase Deficiency patients in 7MM. 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.
The Pyruvate Kinase Deficiency market outlook section of the report helps to build a detailed comprehension of the historic, current and forecasted Pyruvate Kinase Deficiency market trends by analysing the impact of current therapies on the market, unmet needs, drivers, and barriers, and demand for better technology.
The report gives a thorough detail of the Pyruvate Kinase Deficiency 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, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, and view of the key opinion leaders.
Pyruvate Kinase Deficiency Treatment Options
Common Supportive Treatments for Pyruvate Kinase Deficiency include Simple transfusion, Supplemental folic acid and vitamins, Chelation therapy, Phototherapy and Splenectomy.
To redress the current issues, very few companies are currently working toward this indication to meet the needs of the market. Pipeline drugs in clinical development are designed to address the major unmet needs in Pyruvate Kinase Deficiency treatment. The other factors that shall further expedite the growth of the Pyruvate Kinase Deficiency market include increasing awareness about available treatments during the forecast period (2019–2030). A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Pyruvate Kinase Deficiency.
Pyruvate Kinase Deficiency Epidemiology
The Pyruvate Kinase Deficiency epidemiology section covers insights about the historical and current Pyruvate Kinase Deficiency patient pool and forecasted trends for every seven major countries (i.e. the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2017 to 2030.
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 the diagnosed patient pool and their trends along with assumptions undertaken.
Pyruvate Kinase Deficiency Drugs Uptake and Key Market Players
The Pyruvate Kinase Deficiency 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 in the market during the study period. The analysis covers Pyruvate Kinase Deficiency market uptake by drugs; patient uptake by therapies; and sales of each drug.
Report’s Drugs Uptake section helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
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. The global Pyruvate Kinase Deficiency (PKD) treatment market is expected to witness steady growth over the forecast period owing to the increasing availability of novel therapies for Pyruvate Kinase Deficiency (PKD) treatment.
The Pyruvate Kinase Deficiency (PKD) market Size 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 during the forecast period.
Pyruvate Kinase Deficiency Companies:
Agios Pharmaceuticals
Rocket Pharmaceuticals
Centogene AG
Rostock
And many others.
Pyruvate Kinase Deficiency Therapies covered in the report include:
Mitapivat
RP-L301
And many more.
Table of Content
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 is not exhaustive; the final content may vary.
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