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Product Insights
NewLikelihood of Approval and Phase Transition Success Rate Model – ISN-001 in Epidermolysis Bullosa
Empower your strategies with our Likelihood of Approval and Phase Transition Success Rate Model - ISN-001 in Epidermolysis Bullosa report and make more profitable business decisions. This report provides you with the data that allows you to track and predict the specific likelihood of approval (LOA) and phase transition success rate (PTSR) of a drug using GlobalData’s proprietary machine learning algorithms developed using over 10 years of historical data. ISN-001 in Epidermolysis Bullosa Drug Details: ISN-001 (ALLO-ASC) is under development...
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Sector Analysis
Cell and Gene Therapies in Hemophilia A and B – Disease Overview, Treatment Options and Future Market Assessment
Cell and Gene Therapies (CGT) in Hemophilia A and B Market Report Overview The hemophilia A and B CGT market reached $12 million in 2023 with massive expansions across therapeutic areas. The CGT in Hemophilia A and B market research report will aid clients in gaining insight into the competitive landscape of leading CGT agents in Hemophilia A & B including launch date projections, key clinical trial analysis, analyst consensus forecasts, likelihood of approval analysis, and commentary on current and...
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Product Insights
NewHemophilia A (Factor VIII Deficiency) – Drugs In Development, 2024
Empower your strategies with our Hemophilia A (Factor VIII Deficiency) – Drugs In Development, 2024 report and make more profitable business decisions. Hemophilia A is an X-linked recessive disorder caused by deficiency of functional plasma clotting factor VIII (FVIII), which may be inherited or arise from spontaneous mutation. Depending on the level of FVIII activity, patients with hemophilia may present with easy bruising, inadequate clotting of traumatic injury, or in the case of severe hemophilia, spontaneous hemorrhage. Symptoms may include bleeding...
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Product Insights
NewThalassemia – Drugs In Development, 2024
Empower your strategies with our Thalassemia – Drugs In Development, 2024 report and make more profitable business decisions. Thalassemia is a group of inherited blood disorders that affect the body's ability to produce hemoglobin and red blood cells. Symptoms include fussiness, paleness, frequent infections, failure to thrive, poor appetite, and jaundice. Predisposing factors include family history. Treatment includes blood transfusions and bone marrow transplant. The Thalassemia drugs in development market research report provide comprehensive information on the therapeutics under development for...
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Product Insights
NewBeta Thalassaemia – Drugs In Development, 2024
Empower your strategies with our Beta Thalassaemia – Drugs In Development, 2024 report and make more profitable business decisions. Beta-thalassemia is a genetic blood disorder characterized by reduced or absent production of beta-globin chains, a component of hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. This deficiency leads to anemia and various complications. Beta-thalassemia is classified based on the severity of symptoms: In beta-thalassemia major (Cooley's anemia), individuals have little or no beta-globin production, leading...
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Product Insights
NewHemophilia B (Factor IX Deficiency) – Drugs In Development, 2024
Empower your strategies with our Hemophilia B (Factor IX Deficiency) – Drugs In Development, 2024 report and make more profitable business decisions. Hemophilia B is a hereditary bleeding disorder caused by a lack of blood clotting factor IX. Symptoms include bleeding into joints and associated pain and swelling, bruising, excessive bleeding following circumcision, gastrointestinal tract and urinary tract hemorrhage, and nosebleeds. Treatment includes replacing the defective clotting factor. The Hemophilia B (Factor IX Deficiency) drugs in development market research report provide...
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Product Insights
NewAplastic Anemia – Drugs In Development, 2024
Empower your strategies with our Aplastic Anemia – Drugs In Development, 2024 report and make more profitable business decisions. Aplastic anemia is a syndrome of bone marrow failure characterized by peripheral pancytopenia and marrow hypoplasia. Symptoms include fatigue, pale skin, rapid or irregular heart rate, shortness of breath with exertion, nosebleeds and bleeding gums, prolonged bleeding from cuts, skin rash, dizziness, and headache. Risk factors include exposure to toxic chemicals, treatment with high-dose radiation or chemotherapy for cancer, certain blood diseases,...
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Product Insights
NewAcquired (Autoimmune) Hemolytic Anemia – Drugs In Development, 2024
Empower your strategies with our Acquired (Autoimmune) Hemolytic Anemia – Drugs In Development, 2024 report and make more profitable business decisions. Acquired autoimmune hemolytic anemia (AIHA) is a rare blood disorder characterized by the immune system mistakenly attacking and destroying red blood cells. Red blood cells carry oxygen throughout the body, and their destruction leads to anemia (low red blood cell count), causing fatigue, pale skin, and other symptoms. AIHA can be classified into "warm" and "cold" types, depending on the...
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Product Insights
NewGraft Versus Host Disease (GVHD) – Drugs In Development, 2024
Empower your strategies with our Graft Versus Host Disease (GVHD) – Drugs In Development, 2024 report and make more profitable business decisions. Graft-versus-host disease (GVHD) is a complication that can occur after a stem cell or bone marrow transplant in which the newly transplanted donor cells attack the transplant recipient's body. Symptoms include abdominal pain or cramps; nausea, vomiting, and diarrhea; dry or irritated eyes; jaundice; shortness of breath; vaginal dryness; and weight loss. Treatment includes immunosuppressants. The Graft Versus Host...