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Add to CartGlobal Inherited Orphan Blood Disorders Therapeutics Market Growth, Share, Size, Trends and Forecast (2025 - 2031)
By Disorder Type;
Sickle Cell Anemia, Thalassemia and Hereditary Angioedema (HAE).By Therapy Type;
Blood Transfusion, Bone Marrow Transplant, Antibiotics, Analgesics, Hydroxyurea, Iron Chelation, Folic Acid, C1-inhibitors, Kallikrein Inhibitor and Others.By End User;
Hospitals, Pharmacies and Drug Stores.By Geography;
North America, Europe, Asia Pacific, Middle East and Africa and Latin America - Report Timeline (2021 - 2031).Introduction
Global Inherited Orphan Blood Disorders Therapeutics Market (USD Million), 2021 - 2031
In the year 2024, the Global Inherited Orphan Blood Disorders Therapeutics Market was valued at USD 1643.36 million. The size of this market is expected to increase to USD 3004.13 million by the year 2031, while growing at a Compounded Annual Growth Rate (CAGR) of 9.0%.
The global market for therapeutics targeting inherited orphan blood disorders stands at an inflection point, poised for significant growth and transformation in the coming years. Orphan blood disorders, a diverse group of rare hematological conditions, present unique challenges due to their low prevalence and complex pathophysiology. These disorders encompass a spectrum of conditions, including hemoglobinopathies like sickle cell disease and thalassemia, rare coagulation disorders such as hemophilia, and various inherited bone marrow failure syndromes.
Treatment options for inherited orphan blood disorders have been limited, often focusing on symptom management rather than addressing the underlying genetic abnormalities. However, recent advances in biotechnology, including gene therapy, gene editing, and novel pharmacological approaches, have revolutionized the treatment landscape for these conditions. These innovations offer the promise of transformative therapies capable of providing long-term, potentially curative benefits to patients.
One of the key drivers propelling the growth of the global inherited orphan blood disorders therapeutics market is increasing investment in research and development by pharmaceutical companies, academic institutions, and government agencies. The growing understanding of the genetic and molecular basis of these disorders has fueled a surge in drug discovery efforts, leading to the development of a pipeline of promising candidate therapies targeting various aspects of disease pathogenesis.
Regulatory initiatives aimed at incentivizing orphan drug development, such as orphan drug designations and expedited approval pathways, have created a favorable environment for innovation in this space. These regulatory incentives, coupled with a growing emphasis on precision medicine and personalized therapeutics, have spurred interest and investment in the development of targeted therapies tailored to the specific genetic profiles of individual patients.
Despite these promising developments, the market for inherited orphan blood disorders therapeutics still faces challenges, including high treatment costs, limited patient access to innovative therapies, and the need for specialized infrastructure to support advanced treatment modalities such as gene therapy. Moreover, disparities in healthcare access and resources between regions pose additional hurdles to the equitable distribution of novel treatments for rare diseases.
Collaborations between industry stakeholders, patient advocacy groups, and regulatory authorities will be crucial for overcoming these challenges and realizing the full potential of emerging therapies for inherited orphan blood disorders. By fostering an environment of innovation, collaboration, and patient-centered care, the global market for therapeutics targeting these rare hematological conditions is poised to undergo significant expansion, offering hope to patients and caregivers alike.
Global Inherited Orphan Blood Disorders Therapeutics Market Recent Developments
- In February 2021, BioCryst Pharmaceuticals Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending the approval of ORLADEYO (Berotralstat) for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older.
- In February 2021, KalVista reported positive results in its Phase 2 clinical trial for KVD900 as an oral on-demand treatment for HAE attacks.
Segment Analysis
Sickle cell disease, characterized by abnormal hemoglobin molecules in red blood cells, affects millions of people worldwide, particularly those of African, Mediterranean, Middle Eastern, and South Asian descent. The market for sickle cell disease therapeutics is driven by the need for disease-modifying treatments that can alleviate symptoms, prevent complications such as vaso-occlusive crises and organ damage, and ultimately improve patients' quality of life. Current treatment options include hydroxyurea, which can reduce the frequency of painful crises, and newer drugs such as voxelotor and crizanlizumab, which target specific aspects of the disease pathway.
Thalassemia, a group of inherited blood disorders characterized by abnormal hemoglobin production, represents another significant segment of the orphan blood disorders therapeutics market. The severity of thalassemia varies widely, from mild forms that may not require treatment to severe forms that necessitate lifelong transfusions and iron chelation therapy. Emerging therapies such as gene therapy hold promise for addressing the underlying genetic defects in thalassemia, potentially offering curative approaches for some patients. However, challenges related to accessibility, affordability, and long-term safety remain key considerations in the development and adoption of these novel treatments.
Hemophilia, a rare bleeding disorder caused by deficiency or dysfunction of clotting factors, is another important focus of therapeutic development within this market segment. While replacement therapy with clotting factor concentrates has been the mainstay of treatment for decades, newer approaches such as gene therapy aim to provide long-term clotting factor production, potentially reducing the need for frequent infusions and improving patients' quality of life. However, barriers such as high treatment costs, immune responses to gene therapy vectors, and the need for personalized dosing regimens continue to pose challenges to widespread adoption.
In summary, the inherited orphan blood disorders therapeutics market is characterized by a diverse array of conditions, each with its own unique treatment landscape and challenges. Advances in precision medicine, gene therapy, and targeted drug development hold promise for transforming the treatment paradigm for these disorders, but continued investment in research, development, and healthcare infrastructure will be crucial to realizing this potential and improving outcomes for patients worldwide.
Global Inherited Orphan Blood Disorders Therapeutics Segment Analysis
In this report, the Global Inherited Orphan Blood Disorders Therapeutics Market has been segmented by Disorder Type, Therapy Type, End User and Geography.
Global Inherited Orphan Blood Disorders Therapeutics Market, Segmentation by Disorder Type
The Global Inherited Orphan Blood Disorders Therapeutics Market has been segmented by Disorder Type into Sickle Cell Anemia, Thalassemia and Hereditary Angioedema (HAE).
Sickle cell anemia, a hereditary hemoglobin disorder, leads to the production of abnormal hemoglobin molecules, causing red blood cells to become rigid and assume a sickle shape. This deformation results in impaired blood flow, chronic pain, organ damage, and increased susceptibility to infections. Therapeutic interventions for sickle cell anemia aim to alleviate symptoms, prevent complications, and improve quality of life. Recent advancements in gene therapy and targeted pharmacological agents offer promising prospects for disease modification and potential cures.
Thalassemia, another inherited blood disorder, is characterized by abnormal hemoglobin production, leading to inadequate oxygen transport and erythrocyte destruction. Patients with thalassemia may experience severe anemia, fatigue, bone deformities, and organ dysfunction. Management strategies for thalassemia encompass blood transfusions, iron chelation therapy, and hematopoietic stem cell transplantation. Emerging therapies, such as gene editing technologies and novel erythropoiesis-stimulating agents, hold considerable potential for improving outcomes and reducing treatment burden in thalassemia patients.
Hereditary angioedema (HAE) represents a group of rare genetic disorders characterized by recurrent episodes of localized swelling in the skin, gastrointestinal tract, or upper respiratory system. These unpredictable and potentially life-threatening attacks result from deficient or dysfunctional C1 esterase inhibitor protein, leading to unregulated activation of the complement cascade and excessive bradykinin production. Management of HAE involves acute attack treatment with bradykinin receptor antagonists or C1 esterase inhibitor replacement therapy, as well as long-term prophylaxis to reduce attack frequency and severity.
The Global Inherited Orphan Blood Disorders Therapeutics Market continues to evolve with ongoing research efforts aimed at developing innovative treatment modalities, including gene therapy, gene editing, and RNA interference technologies. Collaborative initiatives between pharmaceutical companies, research institutions, and patient advocacy groups are driving progress in understanding the underlying mechanisms of these disorders and advancing therapeutic interventions to improve patient outcomes and quality of life.
Global Inherited Orphan Blood Disorders Therapeutics Market, Segmentation by Therapy Type
The Global Inherited Orphan Blood Disorders Therapeutics Market has been segmented by Therapy Type into Blood transfusion, Bone Marrow Transplant, Antibiotics, Analgesics, Hydroxyurea, Iron Chelation, Folic Acid, C1-inhibitors, kallikrein inhibitor and Others.
Blood transfusion stands as one of the primary therapeutic modalities for managing inherited orphan blood disorders, particularly those characterized by anemia or impaired blood cell production. Transfusion therapy aims to replenish deficient blood components, such as red blood cells or platelets, thereby alleviating symptoms and improving overall health outcomes for patients.
Bone marrow transplant emerges as a potentially curative therapy for certain inherited orphan blood disorders, offering the prospect of replacing dysfunctional or diseased hematopoietic stem cells with healthy donor cells. This approach can be particularly transformative for conditions like sickle cell disease and thalassemia, where the underlying pathology lies in the bone marrow's inability to produce normal blood cells.
Antibiotics play a crucial role in managing inherited blood disorders that predispose patients to recurrent infections, such as severe congenital neutropenia or chronic granulomatous disease. By targeting bacterial pathogens, antibiotics help prevent and treat infections, reducing the risk of complications and improving overall quality of life for affected individuals.
Analgesics represent an essential component of symptom management for inherited blood disorders associated with chronic pain, such as sickle cell disease. These medications help alleviate pain and discomfort, enhancing patients' comfort and functionality while managing the impact of their condition on daily life.
Hydroxyurea has emerged as a cornerstone therapy for conditions like sickle cell disease, offering disease-modifying effects by increasing fetal hemoglobin levels and reducing the frequency of painful crises and other complications. Its widespread use underscores its efficacy in improving outcomes and quality of life for patients with inherited orphan blood disorders.
Iron chelation therapy plays a critical role in managing iron overload, a common complication of conditions such as thalassemia and hereditary hemochromatosis. By removing excess iron from the body, chelation therapy helps prevent organ damage and mitigate the long-term consequences of iron overload, improving patient prognosis and longevity.
Supplementation with folic acid represents a fundamental aspect of supportive care for individuals with certain inherited blood disorders, such as folate deficiency anemia or megaloblastic anemia. Folic acid supplementation helps support red blood cell production and mitigate symptoms associated with folate deficiency, promoting overall health and well-being.
Emerging therapies targeting specific molecular pathways, such as C1-inhibitors and kallikrein inhibitors, hold promise for addressing the underlying pathophysiology of certain inherited blood disorders, particularly those characterized by dysregulated coagulation or complement activation. These targeted approaches offer the potential for more precise and effective treatment, minimizing off-target effects and improving therapeutic outcomes.
In summary, the global market for therapeutics targeting inherited orphan blood disorders encompasses a diverse array of treatment modalities, each tailored to address specific aspects of disease pathology and symptomatology. As our understanding of these disorders continues to evolve and new therapeutic targets emerge, ongoing research and innovation are poised to drive further advancements in the field, ultimately improving outcomes and quality of life for affected individuals.
Global Inherited Orphan Blood Disorders Therapeutics Market, Segmentation by End User
The Global Inherited Orphan Blood Disorders Therapeutics Market has been segmented by End User into Hospitals, Pharmacies and Drug Stores.
The Global Inherited Orphan Blood Disorders Therapeutics Market has undergone segmentation based on end users, categorizing them into hospitals, pharmacies, and drug stores. Hospitals, being the primary healthcare providers, represent a significant portion of the market due to their role in diagnosing and treating patients with inherited orphan blood disorders. These disorders, often rare and complex, require specialized care and expertise, making hospitals the focal point for comprehensive treatment, including diagnosis, therapy initiation, and long-term management. With access to multidisciplinary teams of healthcare professionals and advanced medical technologies, hospitals play a crucial role in delivering integrated care to patients with inherited orphan blood disorders.
Pharmacies also play a vital role in the distribution and management of therapeutics for inherited orphan blood disorders. They serve as crucial points of contact for patients, providing access to prescription medications, specialized therapies, and supportive care products. Pharmacies often collaborate closely with healthcare providers to ensure the timely and accurate dispensing of medications, as well as to provide counseling and education to patients and their caregivers. With their widespread presence in communities and accessibility to patients, pharmacies contribute significantly to the continuum of care for individuals with inherited orphan blood disorders.
Drug stores, which encompass retail outlets specializing in the sale of medications and healthcare products, serve as convenient points of access for patients seeking over-the-counter remedies, nutritional supplements, and supportive care items for managing inherited orphan blood disorders. While drug stores may not offer the same level of specialized care as hospitals or pharmacies, they play a valuable role in meeting the immediate needs of patients, providing convenience and accessibility for essential healthcare products.
Overall, the segmentation of the Global Inherited Orphan Blood Disorders Therapeutics Market by end users reflects the diverse healthcare settings involved in the diagnosis, treatment, and management of these rare and often challenging conditions. By catering to the unique needs of patients through hospitals, pharmacies, and drug stores, healthcare providers can enhance the accessibility, affordability, and effectiveness of therapeutics for inherited orphan blood disorders, ultimately improving patient outcomes and quality of life.
Global Inherited Orphan Blood Disorders Therapeutics Market, Segmentation by Geography
In this report, the Global Inherited Orphan Blood Disorders Therapeutics Market has been segmented by Geography into five regions; North America, Europe, Asia Pacific, Middle East and Africa and Latin America.
Global Inherited Orphan Blood Disorders Therapeutics Market Share (%), by Geographical Region, 2024
The global market for therapeutics targeting inherited orphan blood disorders is characterized by a diverse geographic landscape, with variations in prevalence, treatment accessibility, and healthcare infrastructure across different regions. In North America, including the United States and Canada, the market for inherited orphan blood disorder therapeutics is significant, driven by a combination of factors such as advanced healthcare infrastructure, high prevalence of these disorders, and robust research and development activities. Pharmaceutical companies in this region often focus on developing innovative therapies and leveraging advanced technologies to address the unmet medical needs of patients with inherited orphan blood disorders.
In Europe, countries like the United Kingdom, Germany, France, and Italy are key contributors to the inherited orphan blood disorder therapeutics market. The region benefits from well-established healthcare systems, supportive regulatory frameworks, and a growing emphasis on rare disease research and treatment. Additionally, initiatives from government bodies and patient advocacy groups play a crucial role in raising awareness, improving diagnosis, and enhancing access to therapies for patients with inherited orphan blood disorders.
The Asia Pacific region is witnessing rapid growth in the inherited orphan blood disorder therapeutics market, driven by factors such as increasing healthcare expenditure, expanding patient population, and rising awareness about rare diseases. Countries like China, Japan, India, and South Korea are emerging as key markets, with pharmaceutical companies focusing on developing and commercializing novel therapies tailored to the genetic and clinical characteristics of patients in these regions. However, challenges such as limited healthcare infrastructure in rural areas and regulatory hurdles remain significant barriers to market growth in some parts of the region.
Latin America and the Middle East & Africa region also present opportunities for growth in the inherited orphan blood disorder therapeutics market. While these regions face challenges such as socioeconomic disparities, inadequate healthcare infrastructure, and limited access to specialized care, efforts are underway to improve diagnosis, treatment, and patient support services for individuals with inherited orphan blood disorders. Collaborative initiatives involving governments, healthcare organizations, and pharmaceutical companies are key to overcoming these challenges and expanding access to effective therapies for patients in these regions.
Market Trends
This report provides an in depth analysis of various factors that impact the dynamics of Global Inherited Orphan Blood Disorders Therapeutics Market. These factors include; Market Drivers, Restraints and Opportunities Analysis.
Drivers, Restraints and Opportunity Analysis
Drivers:
- Orphan drug designations
- Precision medicine
- Personalized therapeutics
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Healthcare access -Access to healthcare for inherited orphan blood disorders varies significantly worldwide, influenced by factors such as socioeconomic status, healthcare infrastructure, and government policies. In many regions, especially low- and middle-income countries, access to diagnosis, treatment, and supportive care for these rare conditions remains limited.
One of the primary barriers to healthcare access is the lack of awareness and education among healthcare providers, patients, and communities about inherited orphan blood disorders. Misconceptions and stigma surrounding these conditions can lead to delayed diagnosis, inadequate treatment, and poor health outcomes. Efforts to raise awareness, provide education, and implement screening programs are essential for early detection and intervention.
Healthcare infrastructure plays a crucial role in determining access to specialized care and treatments for inherited orphan blood disorders. Many regions lack specialized centers with expertise in diagnosing and managing these conditions, leading to disparities in access to quality care. Strengthening healthcare systems, training healthcare professionals, and establishing referral networks can help improve access to specialized services for patients with rare blood disorders.
Affordability of healthcare services and treatments is another significant barrier to access, particularly in countries with limited healthcare coverage and high out-of-pocket costs. The high cost of orphan drugs, such as gene therapies and novel biologics, can pose financial burdens on patients and healthcare systems, limiting access to these life-changing treatments. Strategies to improve affordability may include government subsidies, health insurance coverage, and partnerships between pharmaceutical companies and public health agencies to reduce drug prices and expand access programs.
Disparities in access to healthcare services based on geographic location, ethnicity, and socioeconomic status can exacerbate existing inequalities in healthcare access for patients with inherited orphan blood disorders. Addressing these disparities requires a comprehensive approach that considers social determinants of health, promotes equity in healthcare delivery, and engages stakeholders at the local, national, and global levels.
In conclusion, ensuring equitable access to healthcare for patients with inherited orphan blood disorders requires concerted efforts from governments, healthcare providers, pharmaceutical companies, and civil society organizations. By addressing barriers related to awareness, healthcare infrastructure, affordability, and disparities, we can improve diagnosis rates, expand treatment options, and ultimately enhance the quality of life for individuals affected by these rare conditions globally.
Restraints:
- High treatment costs
- Limited patient access
- Specialized infrastructure requirements
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Low prevalence of disorders -The Global Inherited Orphan Blood Disorders Therapeutics Market faces unique challenges due to the low prevalence of these disorders. Inherited orphan blood disorders are rare conditions that affect a relatively small proportion of the population, posing significant hurdles for research, drug development, and commercialization efforts within the pharmaceutical industry. The low prevalence of these disorders often translates to a limited patient pool, making it financially challenging for pharmaceutical companies to invest in the research and development of therapeutics targeting these conditions.
The rarity of inherited orphan blood disorders can lead to underdiagnosis and limited awareness among healthcare providers, potentially delaying the initiation of appropriate treatment and support for affected individuals. The lack of standardized diagnostic criteria and specialized expertise in managing these rare conditions further compounds the challenges faced by patients and healthcare providers alike.
Despite these obstacles, advancements in genetic testing, molecular diagnostics, and precision medicine have enabled improved identification and characterization of inherited orphan blood disorders, facilitating early diagnosis and personalized treatment approaches. Moreover, regulatory incentives such as orphan drug designation and market exclusivity provisions have incentivized pharmaceutical companies to pursue research and development initiatives targeting rare diseases, including inherited orphan blood disorders.
Collaborative efforts between academia, industry, patient advocacy groups, and regulatory agencies have also played a pivotal role in advancing research, raising awareness, and improving access to therapeutics for individuals with inherited orphan blood disorders. By fostering partnerships and leveraging collective expertise and resources, stakeholders across the healthcare ecosystem can work together to address the unmet needs of patients with rare diseases and drive innovation in the development of novel therapeutics and treatment modalities.
Overall, while the low prevalence of inherited orphan blood disorders presents formidable challenges for stakeholders in the therapeutics market, concerted efforts aimed at increasing disease awareness, advancing research, and fostering collaboration hold promise for improving outcomes and quality of life for affected individuals. By prioritizing patient-centered approaches and advocating for policies that support innovation and access to care, the healthcare community can strive to address the unique needs of this vulnerable patient population.
Opportunities:
- Biomarker identification
- Patient access initiatives
- Global partnerships
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Research and development -Research and development (R&D) in the global inherited orphan blood disorders therapeutics market play a pivotal role in driving innovation, advancing treatment options, and improving patient outcomes. Pharmaceutical companies, biotechnology firms, and academic institutions are actively engaged in R&D activities aimed at discovering novel therapies, elucidating disease mechanisms, and developing targeted interventions for various inherited orphan blood disorders.
One significant area of R&D focus is the development of gene therapies for inherited orphan blood disorders. Gene therapy holds immense promise for treating genetic diseases by correcting or replacing defective genes responsible for the disorder. Recent advancements in gene editing technologies, such as CRISPR-Cas9, have propelled the development of gene therapies targeting disorders like hemophilia, sickle cell disease, and thalassemia. Clinical trials evaluating the safety and efficacy of gene therapy approaches are underway globally, with encouraging results reported in some cases.
Another area of active R&D is the exploration of novel pharmacological interventions and therapeutic modalities for inherited orphan blood disorders. This includes the development of small molecule drugs, monoclonal antibodies, RNA-based therapies, and cell-based therapies targeting specific disease pathways or cellular mechanisms implicated in these disorders. Researchers are also investigating innovative treatment strategies, such as gene silencing techniques and gene editing platforms, to modulate gene expression and alleviate disease symptoms.
R&D efforts in the inherited orphan blood disorders therapeutics market extend to the development of supportive care interventions and symptom management strategies to improve the quality of life for patients. This includes the development of adjunctive therapies, supportive treatments for complications associated with these disorders, and interventions to address comorbidities and disease-related complications.
Collaboration and partnership between industry stakeholders, academia, patient advocacy groups, and regulatory agencies are essential to accelerate R&D progress, facilitate knowledge exchange, and overcome challenges in drug development and commercialization. Additionally, initiatives aimed at improving patient recruitment for clinical trials, enhancing research infrastructure, and fostering innovation ecosystems contribute to advancing the field of inherited orphan blood disorders therapeutics.
Overall, ongoing R&D efforts underscore the commitment of the global healthcare community to addressing the unmet medical needs of patients with inherited orphan blood disorders, with the ultimate goal of developing safe, effective, and accessible therapies to improve patient outcomes and quality of life.
Competitive Landscape Analysis
Key players in Global Inherited Orphan Blood Disorders Therapeutics Market include.
- Biogen Idec
- amida Cell
- Anthera Pharmaceuticals
- Alnylam Pharmaceuticals
- Sangamo BioSciences
- Global Blood Therapeutics
- Mast Therapeutics
- Genetix Pharmaceuticals Inc
In this report, the profile of each market player provides following information:
- Company Overview and Product Portfolio
- Key Developments
- Financial Overview
- Strategies
- Company SWOT Analysis
- Introduction
- Research Objectives and Assumptions
- Research Methodology
- Abbreviations
- Market Definition & Study Scope
- Executive Summary
- Market Snapshot, By Disorder Type
- Market Snapshot, By Therapy Type
- Market Snapshot, By End User
- Market Snapshot, By Region
- Global Inherited Orphan Blood Disorders Therapeutics Market Dynamics
- Drivers, Restraints and Opportunities
- Drivers
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Orphan drug designations
-
Precision medicine
-
Personalized therapeutics
-
Healthcare access
-
- Restraints
-
High treatment costs
-
Limited patient access
-
Specialized infrastructure requirements
-
Low prevalence of disorders
-
- Opportunities
-
Biomarker identification
-
Patient access initiatives
-
Global partnerships
-
Research and development
-
- Drivers
- PEST Analysis
- Political Analysis
- Economic Analysis
- Social Analysis
- Technological Analysis
- Porter's Analysis
- Bargaining Power of Suppliers
- Bargaining Power of Buyers
- Threat of Substitutes
- Threat of New Entrants
- Competitive Rivalry
- Drivers, Restraints and Opportunities
- Market Segmentation
- Global Inherited Orphan Blood Disorders Therapeutics Market, By Disorder Type, 2021-2031 (USD Million)
- Sickle Cell Anemia
- Thalassemia
- Hereditary Angioedema (HAE)
- Global Inherited Orphan Blood Disorders Therapeutics Market, By Therapy Type, 2021-2031 (USD Million)
- Blood transfusion
- Bone Marrow Transplant
- Antibiotics
- Analgesics
- Hydroxyurea
- Iron Chelation
- Folic Acid
- C1-inhibitors
- kallikrein inhibitor
- Others
- Global Inherited Orphan Blood Disorders Therapeutics Market, By End User, 2021-2031 (USD Million)
- Hospitals
- Pharmacies
- Drug Stores
- Global Inherited Orphan Blood Disorders Therapeutics Market, By Geography, 2021-2031 (USD Million)
- North America
- United States
- Canada
- Europe
- Germany
- United Kingdom
- France
- Italy
- Spain
- Nordic
- Benelux
- Rest of Europe
- Asia Pacific
- Japan
- China
- India
- Australia & New Zealand
- South Korea ASEAN (Association of South East Asian Countries)
- Rest of Asia Pacific
- Middle East & Africa
- GCC
- Israel
- South Africa
- Rest of Middle East & Africa
- Latin America
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- North America
- Global Inherited Orphan Blood Disorders Therapeutics Market, By Disorder Type, 2021-2031 (USD Million)
- Competitive Landscape
- Company Profiles
- Biogen Idec
- amida Cell
- Anthera Pharmaceuticals
- Alnylam Pharmaceuticals
- Sangamo BioSciences
- Global Blood Therapeutics
- Mast Therapeutics
- Genetix Pharmaceuticals Inc
- Company Profiles
- Analyst Views
- Future Outlook of the Market