Takeda

This study looks back at medical records of 94 people with a rare inherited blood disorder called cTTP. These people received a lab-made version of a missing enzyme (rADAMTS13) to prevent or treat dangerous blood clots. The goal is to see how the treatment affected their health, …

This study monitors the safety and effectiveness of Takhzyro, an approved drug for hereditary angioedema (HAE), in teenagers and adults in South Korea. HAE causes sudden swelling under the skin or inside the body. About 35 participants will be followed during their regular medica…

This study tests an experimental drug called TAK-226 in 27 Japanese adults with a bone marrow condition called myelodysplastic syndromes (MDS) who need regular blood transfusions. The goal is to see if TAK-226 can help them go without transfusions for at least 8 weeks. Participan…

This study tests a new drug called zasocitinib in 20 adults with active Crohn's disease who haven't responded to other treatments. The goal is to see if it's safe and how it works in the body. Participants take the drug for 12 weeks and have a colonoscopy to check the gut.

This survey studies how well people with Gaucher disease, their caregivers, and home nurses understand educational materials for VPRIV home infusions. About 60 participants from European countries will fill out a questionnaire. The goal is to improve safety and ease of home treat…

This study will collect information from medical records and routine doctor visits of 100 people with hereditary angioedema (HAE) types 1 or 2 in Egypt. The goal is to understand how the condition is diagnosed and treated in everyday medical practice. No new treatments are being …

This study looks at the safety of a drug called Adzynma in people with a rare inherited blood disorder (cTTP) that causes dangerous blood clots. Researchers will review medical records of about 50 children and adults who have already received Adzynma in regular care. The main goa…