Therapeutic Focus - Rare Diseases | Takeda Pharmaceuticals
Rare Diseases
We aspire to transform the treatment of rare diseases in hematology and hemostasis, immunology, metabolic and lysosomal storage disorders, and cytomegalovirus (CMV) infection in transplant.
These rare diseases can have symptoms that vary widely and progress very differently from person to person, which means that people affected by these diseases are frequently misdiagnosed, leading to significant diagnostic delay.
Rare Hematology and Hemostasis
We are a leader with more than 70 years of experience in rare hematology. Our experience in hematology means we are well-prepared to meet today’s needs as we pursue future developments in the treatment of bleeding disorders. Together with the hematology community, we are raising expectations for the future, including earlier diagnosis, earlier and protection against bleeds, and more personalized patient care.
Disease areas
Hemophilia
Von Willebrand Disease
Thrombotic thrombocytopenic purpura
Rare Immunology
Within Rare Immunology we focus on driving continuous innovation and personalized care through our portfolio of plasma products and innovative targeted treatments, devices, diagnostics and other technological services, for the benefit of patients with rare immunological disorders. Our core therapeutic areas include Hereditary Angioedema, immuno-deficiencies, rare autoimmune disorders, post-transplant complications and rare specialty care.
Disease areas
Hereditary Angioedema
Immune Deficiency Diseases
Rare Autoimmune Disorders
Hypoalbuminemia and Hypovolemia
Severe Congenital Protein C Deficiency
Prothrombin Deficiency
Alpha-1 Antitrypsin Deficiency
Rare Metabolic
We have a strong legacy in developing treatments for lysosomal storage disorders (LSDs), with a portfolio that includes commercial products, late-stage investigational therapies, and pipeline candidates. Because rare genetic and metabolic diseases can have symptoms that vary widely and progress differently from person to person, we empower global education and awareness, and partner with medical and research organizations. We are committed to helping reduce the amount of time between the onset of symptoms and diagnosis and to accelerating the development of innovative new treatments.
Select disease areas
Hunter Syndrome
Fabry Disease
Gaucher Disease
CMV and Transplant