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Primary Ciliary Dyskinesia - Market Insights, Epidemiology and Market Forecast - 2028

  • ID: 4845158
  • Drug Pipelines
  • September 2019
  • Region: Global
  • 213 pages
  • DelveInsight
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FEATURED COMPANIES

  • Eloxx Pharmaceuticals
  • Santhera Pharmaceuticals
  • Synspira Therapeutics
  • Translate Bio
  • Vertex Pharmaceuticals
  • MORE
Primary Ciliary Dyskinesia - Market Insights, Epidemiology and Market Forecast-2028 report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Primary Ciliary Dyskinesia in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom) and Japan.

The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Primary Ciliary Dyskinesia (PCD) from 2017 to 2028 segmented by seven major markets. The Report also covers current treatment practice, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.

Geography Covered
  • The United States
  • EU5 (Germany, France, Italy, Spain and the United Kingdom)
  • Japan
Study Period:2017-2028

Primary Ciliary Dyskinesia - Disease Understanding and Treatment Algorithm

The Primary Ciliary Dyskinesia (PCD) market report gives the thorough understanding of the Primary Ciliary Dyskinesia (PCD) by including details such as disease definition, causes, symptoms, pathophysiology, and diagnostic trends. It also provides overview of treatment and management of the disease in the US, EU5 and Japan.

Primary ciliary dyskinesia (PCD) is an autosomal recessive genetic disorder caused by the impaired function of cilia of the respiratory tract epithelial cells, which may also involve other ciliated cells in the reproductive system of the male or female.

Primary Ciliary Dyskinesia Epidemiology

The Primary Ciliary Dyskinesia epidemiology division provide the insights about historical and current patient pool and forecasted trend for every seven 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 report also provides the diagnosed patient pool and the prevalence based on clinical manifestations in the US, EU5 and Japan.

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (total diagnosed prevalent cases, and prevalence based on clinical manifestations) scenario of Primary Ciliary Dyskinesia in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017-2028.

According to this report, total prevalent population of Primary Ciliary Dyskinesia in seven major markets was found to be 80,815 in 2017.

Primary Ciliary Dyskinesia Drug Chapters

This segment of the PCD Drug report encloses the detailed analysis of the emerging drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, and the latest news and press releases.

At present, therapeutic approach of PCD involves symptomatic treatment. The PCD market forecast provided in the report solely focuses on the market revenue generated by the off-label systemic therapies, such as antibiotics, inhaled corticosteroids, rhDNase and bronchodilators. Till date no drug has obtained approval for the treatment of PCD. Detailed chapter for upcoming therapies like Vertex Pharmaceuticals novel inhibitor of the epithelial sodium channel (ENaC) VX-371, and other promising therapies such as POL6014 (Santhera Pharmaceuticals) have been covered in the report.

Primary Ciliary Dyskinesia (PCD) Market Outlook

The PCD market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers.

This segment gives a through detail of emerging therapies and other promising therapies by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria's, 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 market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to this report, the market of Primary Ciliary Dyskinesia (PCD) in 7MM was found to be USD 490.66 million in 2017.

Primary Ciliary Dyskinesia (PCD) Drugs Uptake

This section focusses on the rate of uptake of the potential drugs that will get launched in the market during the study period from 2017-2028. The analysis covers market uptake by drugs; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows 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.

Primary Ciliary Dyskinesia (PCD) Report Insights
  • Patient Population
  • Therapeutic Approaches
  • Pipeline Analysis
  • Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies
Primary Ciliary Dyskinesia (PCD) Report Key Strengths
  • 10 Year Forecast
  • 7MM Coverage
  • Epidemiology Segmentation
  • Drugs Uptake
  • Market Size by Therapies
  • Key Cross Competition
Primary Ciliary Dyskinesia (PCD) Report Assessment
  • Pipeline Product Profiles
  • Key Products and Key Players
  • Market Drivers and Barriers
Key Benefits
  • This report will help to develop Business Strategies by understanding the trends shaping and driving Primary Ciliary Dyskinesia (PCD) market.
  • Organize sales and marketing efforts by identifying the best opportunities for Primary Ciliary Dyskinesia (PCD) market.
  • To understand the future market competition in the Primary Ciliary Dyskinesia (PCD) market.
Note: Product cover images may vary from those shown
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FEATURED COMPANIES

  • Eloxx Pharmaceuticals
  • Santhera Pharmaceuticals
  • Synspira Therapeutics
  • Translate Bio
  • Vertex Pharmaceuticals
  • MORE
1. Key Insights

2. Primary Ciliary Dyskinesia: Epidemiology Overview at a Glance

3. Primary Ciliary Dyskinesia: Market Overview at a Glance
3.1 Total Market Share (%) Distribution of Primary Ciliary Dyskinesia in 2017
3.2 Total Market Share (%) Distribution of Primary Ciliary Dyskinesia in 2028

4. Primary Ciliary Dyskinesia (PCD): Disease Background and Overview
4.1 Introduction
4.2 Causes
4.3 Risk Factors
4.4 Signs and Symptoms

5. Inheritance pattern
5.1 Disease Manifestations
5.2 Genetics
5.3 Pathophysiology
5.3.1 Structure and function of cilia
5.3.2 Ciliary ultrastructural defects
5.3.3 Proposed Pathogenesis of Primary Ciliary Dyskinesia Lung Disease
5.4 Related Disorders
5.5 Diagnostic Approach
5.5.1 Diagnostic Tests
5.5.2 Recommended PCD Diagnostic Criteria by Age
5.5.3 Tests Not Recommended for PCD Diagnosis
5.5.4 European Respiratory Society guidelines for the diagnosis of primary ciliary dyskinesia
5.6 North American Primary Ciliary Dyskinesia (PCD) Foundation guidelines
5.7 A comparison between the European Respiratory Society (ERS) and North American primary ciliary dyskinesia (PCD) Foundation guidelines
5.8 American Thoracic Society (ATS) Guideline Aids in Diagnosing Primary Ciliary Dyskinesia
5.8.1 Primary Ciliary Dyskinesia (PCD) Management
5.8.2 Diagnostic algorithm for Primary Ciliary Dyskinesia
5.9 Future Prospects of Primary Ciliary Dyskinesia Diagnosis

6. Treatment
6.1 Treatment Algorithm
6.2 Manifestation-wise Treatment
6.2.1 Respiratory Manifestations Treatment
6.2.2 Ear, nose and throat problems in PCD
6.2.3 Other Systems

7. Unmet Needs

8. Organizations contributing toward PCD

9. KOL’s Views: Primary Ciliary Dyskinesia

10. Key Cross Competitors

11. Emerging Therapies
11.1 VX-371: Vertex Pharmaceuticals
11.1.1 Product Description
11.1.2 Other development Activities
11.1.3 Clinical Development
11.1.4 Product Profile
11.2 POL6014: Santhera Pharmaceuticals
11.2.1 Product Description
11.2.2 Other development Activities
11.2.3 Product Profile
11.3 Primary Ciliary Dyskinesia Research Program: Translate Bio
11.3.1 Product Description
11.3.2 Product Profile
11.4 SNSP113: Synspira Therapeutics
11.4.1 Product Description
11.4.2 Other development Activities
11.4.3 Clinical Development
11.4.4 Product Profile
11.5 ELX-02: Eloxx Pharmaceuticals
11.5.1 Product Description
11.5.2 Other development Activities
11.5.3 Product Profile
11.6 Future Treatment Therapies for Primary Ciliary Dyskinesia

12. Epidemiology and Patient Population
12.1 Key Findings

13. 7MM Total Prevalent Patient Population of PCD

14. Country Wise-Epidemiology of Primary Ciliary Dyskinesia
14.1 United States
14.1.1 Assumptions and Rationale
14.1.2 Total Prevalent Population of Primary Ciliary Dyskinesia
14.1.3 Total Diagnosed Population of Primary Ciliary Dyskinesia
14.1.4 Clinical Manifestations of Primary Ciliary Dyskinesia
14.2 EU5 Countries
14.2.1 Assumptions and Rationale
14.3 Germany
14.3.1 Total Prevalent Population of Primary Ciliary Dyskinesia
14.3.2 Total Diagnosed Population of Primary Ciliary Dyskinesia
14.3.3 Clinical Manifestations of Primary Ciliary Dyskinesia
14.4 France
14.4.1 Total Prevalent Population of Primary Ciliary Dyskinesia
14.4.2 Total Diagnosed Population of Primary Ciliary Dyskinesia
14.4.3 Clinical Manifestations of Primary Ciliary Dyskinesia
14.5 Italy
14.5.1 Total Prevalent Population of Primary Ciliary Dyskinesia
14.5.2 Total Diagnosed Population of Primary Ciliary Dyskinesia
14.5.3 Clinical Manifestations of Primary Ciliary Dyskinesia
14.6 Spain
14.6.1 Total Prevalent Population of Primary Ciliary Dyskinesia
14.6.2 Total Diagnosed Population of Primary Ciliary Dyskinesia
14.6.3 Clinical Manifestations of Primary Ciliary Dyskinesia
14.7 United Kingdom
14.7.1 Total Prevalent Population of Primary Ciliary Dyskinesia
14.7.2 Total Diagnosed Population of Primary Ciliary Dyskinesia
14.7.3 Clinical Manifestations of Primary Ciliary Dyskinesia
14.8 Japan
14.8.1 Assumptions and Rationale
14.8.2 Total Prevalent Population of Primary Ciliary Dyskinesia
14.8.3 Total Diagnosed Population of Primary Ciliary Dyskinesia
14.8.4 Clinical Manifestations of Primary Ciliary Dyskinesia

15. Case-Reports
15.1 Diagnostic Journey of a girl who couldn't stop coughing
15.2 Early Lung Disease in Young Children with Primary Ciliary Dyskinesia - Therapeutic Perspective
15.3 Ultrastructural pathology of primary ciliary dyskinesia: report about 125 cases in Germany - Diagnostic Perspective
15.4 Individualized physical training in the therapy of Primary Ciliary Dyskinesia - A case report - Preventive Therapy Perspective
15.5 Kartagener’s syndrome
15.6 Nasal nitric oxide is low early in life: case study of two infants with primary ciliary dyskinesia

16. Projects aimed to push the research of Primary Ciliary Dyskinesia forward
16.1 BESTCILIA Project (Better Experimental Screening and Treatment for Primary Ciliary Dyskinesia) - December 2012 - May 2016 (Duration 42 months)
16.1.1 Executive Summary
16.1.2 Project Context and Objectives
16.1.3 Project Results
16.1.4 Potential Impact
16.1.5 Dissemination of project results - discourse with the scientific community
16.1.6 Funding
16.2 BEAT - PCD (Better Experimental Approaches to Treat Primary Ciliary Dyskinesia)
16.2.1 Introduction
16.2.2 Objectives
16.2.3 Proceedings of the COST action BM1407 inaugural conference BEAT-PCD: translational research in primary ciliary dyskinesia - bench, bedside, and population perspectives - Summary

17. Primary Ciliary Dyskinesia: 7 Major Market Analysis
17.1 Key Findings
17.2 Market Size of Primary Ciliary Dyskinesia in 7MM
17.2.1 Total Market Size of Primary Ciliary Dyskinesia
17.2.2 Market Size of Primary Ciliary Dyskinesia by Therapies

18. United States Market Outlook
18.1.1 United States Market Size
18.1.2 Total Market Size of Primary Ciliary Dyskinesia
18.1.3 Market Size of Primary Ciliary Dyskinesia by Therapies

19. EU-5 Countries: Market Outlook
19.1 Germany
19.1.1 Total Market size of Primary Ciliary Dyskinesia
19.1.2 Market Size of Primary Ciliary Dyskinesia by Therapies
19.2 France
19.2.1 Total Market Size of Primary Ciliary Dyskinesia
19.2.2 Market Size of Primary Ciliary Dyskinesia by Therapies
19.3 Italy
19.3.1 Total Market Size of Primary Ciliary Dyskinesia
19.3.2 Market Size of Primary Ciliary Dyskinesia by Therapies
19.4 Spain
19.4.1 Total Market Size of Primary Ciliary Dyskinesia
19.4.2 Market Size of Primary Ciliary Dyskinesia by Therapies
19.5 United Kingdom
19.5.1 Total Market Size of Primary Ciliary Dyskinesia
19.5.2 Market Size of Primary Ciliary Dyskinesia by Therapies

20. Japan: Market Outlook
20.1 Total Market Size of Primary Ciliary Dyskinesia
20.2 Market Size of Primary Ciliary Dyskinesia by Therapies

21. Market Drivers

22. Market Barriers

23. Appendix
23.1 Report Methodology

24. Capabilities

25. Disclaimer

26. About the Publisher
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  • Vertex Pharmaceuticals
  • Santhera Pharmaceuticals
  • Translate Bio
  • Synspira Therapeutics
  • Eloxx Pharmaceuticals
Note: Product cover images may vary from those shown
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