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Inherited Orphan Blood Disorders Therapeutics Market to 2019 - Breakthrough Drugs Remain Elusive Against Backdrop of High Unmet Need

, in-depth profiling of major products and details of recent industry deals.

The report is built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis by GBI Research's team of industry experts.

The sickle cell anemia market will be the fastest growing of the three markets during the forecast period with a Compound Annual Growth Rate (CAGR) of 9% seeing it reach $70m in 2019. The thalassemia market will grow at a lower CAGR of 7% to reach $59m in 2019. HAE represents the largest but slowest growing market, expected to reach $1.89 billion by 2019 at a CAGR of 3%.



- Current and future treatment trends for three rare diseases

- Market forecasts for three rare diseases across seven major markets

- Analysis of the developmental pipelines for three rare diseases

- Information on recent industry deals


Reasons to buy

- Understand how the inherited orphan blood diseases market has developed and is continuing to develop within the featured markets

- Predict which products are likely to be most successful in the future

- Gain an understanding of the possible market available for specific products


Table of Contents

1 Table of Contents 4

1.1 List of Tables 6

1.2 List of Figures 6

2 Inherited Orphan Blood Diseases - Introduction 8

2.1 Overview 8

3 Inherited Orphan Blood Disorders - Therapeutic Landscape 9

3.1 Sickle Cell Anemia 9

3.1.1 Disease Overview 9

3.1.2 Revenues 14

3.1.3 Revenue Analysis by Country 14

3.1.4 Annual Cost of Therapy 18

3.1.5 Treatment Usage Patterns 19

3.1.6 Marketed Products 20

3.1.7 Market Drivers 21

3.1.8 Market Restraints 21

3.2 Sickle Cell Anemia: Research and Development Pipeline 22

3.2.1 Overview 22

3.2.2 Pipeline by Clinical Phase of De

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Related medicine technology :

1. Savara Pharmaceuticals AeroVanc Granted U.S. Orphan Drug Designation for the Treatment of MRSA Lung Infection in Cystic Fibrosis Patients
2. FDA Grants Orphan Drug Status To Morphoteks Amatuximab (MORAb-009)
3. Albireo Has Been Granted Orphan Drug Designation for A4250 from the US FDA for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) and for Primary Biliary Cirrhosis (PBC)
4. EMA Grants Orphan Designation to Edison Pharmaceuticals for EPI-743 for Treatment of Leigh Syndrome
5. FDA Grants Orphan Drug Designation To GeNO LLC For Use Of Inhaled Nitric Oxide In Treatment Of Persistant Pulmonary Hypertension Of The Newborn (PPHN)
6. Albireo Receives Positive Opinion for Orphan Drug Designation for A4250 for Severe Liver Diseases
7. Grifols Initiates Safety Study of Inhaled Alpha1-Proteinase Inhibitor (Human) Following Orphan Drug Designation for Cystic Fibrosis
8. European Commission Grants Orphan Medicinal Product Designation for Emmaus Medicals Sickle Cell Treatment
9. CSL Behring Receives FDA Orphan Drug Designation for rIX-FP, a Novel Therapy in Development to Treat Congenital Factor IX Deficiency (Hemophilia B)
10. Tris Pharma Announces Approval of First-Ever Generic to Delsym® Dextromethorphan Polistirex Extended-Release Suspension
11. Perrigo Announces FDA Final Approval Of Tris Pharmas Dextromethorphan Polistirex Extended-Release Oral Suspension
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