Sections
| Title | Starting Page | Number of Pages |
|---|
| Table of Contents | 4 | 4 |
| List of Tables | 6 | 1 |
| List of Figures | 6 | 2 |
| Inherited Orphan Blood Diseases - Introduction | 8 | 1 |
| Overview | 8 | 1 |
| Inherited Orphan Blood Disorders - Therapeutic Landscape | 9 | 63 |
| Sickle Cell Anemia | 9 | 1 |
| Disease Overview | 9 | 1 |
| Etiology | 9 | 2 |
| Epidemiology | 11 | 1 |
| Symptoms | 12 | 1 |
| Treatment | 13 | 1 |
| Revenues | 14 | 1 |
| Revenue Analysis by Country | 14 | 1 |
| The US | 14 | 1 |
| The UK | 15 | 1 |
| France | 15 | 1 |
| Germany | 16 | 1 |
| Italy | 16 | 1 |
| Spain | 17 | 1 |
| Japan | 17 | 1 |
| Annual Cost of Therapy | 18 | 1 |
| Treatment Usage Patterns | 19 | 1 |
| Diseased Population | 19 | 1 |
| Diagnosed Population | 19 | 1 |
| Treated Population | 19 | 1 |
| Marketed Products | 20 | 1 |
| Hydroxyurea | 20 | 1 |
| Other Treatments | 20 | 1 |
| Market Drivers | 21 | 1 |
| High Unmet Need | 21 | 1 |
| Low Market Competition | 21 | 1 |
| High Treatment Seeking Rate | 21 | 1 |
| Growth of Diseased Population | 21 | 1 |
| Market Restraints | 21 | 1 |
| Absence of Approved Disease Modifying Treatments | 21 | 1 |
| Small Patient Population | 21 | 1 |
| Sickle Cell Anemia: Research and Development Pipeline | 22 | 1 |
| Overview | 22 | 1 |
| Pipeline by Clinical Phase of Development | 23 | 1 |
| Discovery | 23 | 1 |
| Pre-Clinical | 23 | 1 |
| Phase I | 24 | 1 |
| Phase II | 25 | 2 |
| Phase III | 27 | 1 |
| Trends in Sickle Cell Anemia Pipeline | 28 | 2 |
| Promising Pipeline Molecules | 30 | 1 |
| HQK-1001 | 30 | 1 |
| Panobinostat | 31 | 1 |
| ANX-188 | 32 | 1 |
| Key Takeaway | 32 | 1 |
| Thalassemia | 33 | 1 |
| Disease Overview | 33 | 1 |
| Etiology | 33 | 2 |
| Epidemiology | 35 | 1 |
| Symptoms | 36 | 1 |
| Treatment | 37 | 1 |
| Revenues | 38 | 1 |
| Revenue Analysis by Country | 39 | 1 |
| The US | 39 | 1 |
| The UK | 39 | 1 |
| France | 40 | 1 |
| Germany | 40 | 1 |
| Italy | 41 | 1 |
| Spain | 41 | 1 |
| Japan | 42 | 1 |
| Annual Cost of Therapy | 43 | 1 |
| Treatment Usage Patterns | 44 | 1 |
| Diseased Population | 44 | 1 |
| Diagnosed Population | 44 | 1 |
| Treated Population | 44 | 1 |
| Marketed Products | 44 | 1 |
| Desferal | 44 | 1 |
| Exjade | 45 | 1 |
| Ferriprox | 45 | 1 |
| Other Treatments | 45 | 1 |
| Market Drivers | 46 | 1 |
| High Unmet Need | 46 | 1 |
| Low Market Competition | 46 | 1 |
| Population Specificity | 46 | 1 |
| Growth of Diseased Population | 46 | 1 |
| Market Restraints | 46 | 1 |
| Absence of Approved Disease-Modifying Treatments | 46 | 1 |
| Small Patient Population | 46 | 1 |
| Cumbersome route of administration and frequency of dosing | 46 | 1 |
| Thalassemia - Research and Development Pipeline | 47 | 1 |
| Overview | 47 | 1 |
| Pipeline by Clinical Phase of Development | 48 | 1 |
| Discovery | 48 | 1 |
| Pre-Clinical | 48 | 1 |
| Phase I | 48 | 2 |
| Phase II | 50 | 1 |
| Phase III | 51 | 1 |
| Trends in Thalassemia Pipeline | 51 | 2 |
| Promising Pipeline Molecules | 53 | 1 |
| Sotatercept | 53 | 1 |
| ACE-536 | 53 | 1 |
| HQK-1001 | 54 | 1 |
| Key Takeaway | 54 | 1 |
| Hereditary Angioedema | 55 | 1 |
| Disease Overview | 55 | 1 |
| Etiology | 55 | 1 |
| Epidemiology | 55 | 1 |
| Symptoms | 55 | 1 |
| Treatment | 55 | 1 |
| Revenues | 56 | 1 |
| Revenue Analysis by Country | 57 | 1 |
| The US | 57 | 1 |
| The UK | 57 | 1 |
| France | 58 | 1 |
| Germany | 58 | 1 |
| Italy | 59 | 1 |
| Spain | 59 | 1 |
| Japan | 60 | 1 |
| Annual Cost of Therapy | 61 | 1 |
| Treatment Usage Patterns | 62 | 1 |
| Diseased Population | 62 | 1 |
| Diagnosed Population | 62 | 1 |
| Treated Population | 62 | 1 |
| Marketed Products | 63 | 1 |
| Cinryze | 63 | 1 |
| Kalbitor | 63 | 1 |
| Market Drivers | 64 | 1 |
| Novel Therapies | 64 | 1 |
| Disease Awareness Programs | 64 | 1 |
| Orphan Drug Exclusivity | 64 | 1 |
| Market Restraints | 64 | 1 |
| Low Rate/Delayed Diagnosis | 64 | 1 |
| Small Patient Population | 64 | 1 |
| Hereditary Angioedema: Research and Development Pipeline | 65 | 1 |
| Overview | 65 | 1 |
| Pipeline by Clinical Phase of Development | 66 | 1 |
| Discovery | 66 | 1 |
| Pre-Clinical | 66 | 1 |
| Phase I | 67 | 1 |
| Phase II | 67 | 1 |
| Phase III | 68 | 1 |
| Trends in Hereditary Angioedema Pipeline | 69 | 2 |
| Promising Pipeline Molecules | 71 | 1 |
| Key Takeaway | 71 | 1 |
| Inherited Orphan Blood Disorders Therapeutics - Strategic Consolidations | 72 | 4 |
| Segmentation by Year | 72 | 1 |
| Segmentation by Indication | 72 | 1 |
| Segmentation by Geography | 73 | 1 |
| Segmentation by Deal Value | 73 | 1 |
| Segmentation by Deal Type | 74 | 1 |
| Major Mergers and Acquisitions | 74 | 1 |
| Major Licensing Agreements | 75 | 1 |
| Inherited Orphan Blood Disorders Therapeutics - Appendix | 76 | 6 |
| Market Definitions | 76 | 1 |
| Abbreviations | 76 | 1 |
| Sources | 76 | 1 |
| Research Methodology | 77 | 5 |
| Coverage | 77 | 1 |
| Secondary Research | 77 | 1 |
| Primary Research | 78 | 1 |
| Therapeutic Landscape | 78 | 1 |
| Epidemiology-based Forecasting | 79 | 1 |
| Market Size by Geography | 80 | 1 |
| Geographical Landscape | 81 | 1 |
| Pipeline Analysis | 81 | 1 |
| Competitive Landscape | 81 | 1 |
| Expert Panel Validation | 81 | 1 |
| Contact Us | 81 | 1 |
| Disclaimer | 81 | 1 |
