HEMOPHILIA A

This study is observing 427 people with hemophilia A over four years to see if long-term treatment with Elocta helps protect their joints from damage. Participants are already using Elocta as part of their regular care. The main goal is to see if this treatment reduces joint blee…

This early-stage study is testing a one-time gene therapy called BAY2599023 (DTX201) for adults with severe hemophilia A. The therapy uses a modified virus to deliver a healthy copy of the gene needed to make clotting Factor VIII, with the goal of helping the body produce this pr…

This is an early safety study testing a new gene therapy approach for adults with severe hemophilia A, a serious bleeding disorder. Researchers will collect a patient's own blood stem cells, genetically modify them in a lab to produce the missing clotting factor inside platelets,…

This study is testing a single-dose gene therapy called valoctocogene roxaparvovec (BMN 270) for Japanese adults with severe hemophilia A. The goal is to see if this one-time intravenous treatment can help the body produce its own blood-clotting factor, reducing or eliminating th…

This study is monitoring the long-term safety of ADYNOVI/ADYNOVATE, a medication for hemophilia A, in 207 patients who are already using it as part of their regular care. The main goal is to check for any side effects that might develop over time, especially those affecting the k…

This study aims to track the long-term safety and durability of a one-time gene therapy (BMN 270) for severe Hemophilia A over approximately 10 years. It will follow about 172 patients who received the therapy in earlier BioMarin trials. The main goal is to see if the treatment's…

This study aims to monitor the long-term safety of Jivi, an approved medication for hemophilia A, over at least four years. It will follow 62 patients who are already receiving Jivi regularly to prevent bleeding episodes. Researchers will collect health data during patients' rout…

This study aims to see how well a new treatment, efanesoctocog alfa, works over five years to protect the joints of people with Hemophilia A. It will follow 200 patients in the US and Japan who are already receiving this treatment as part of their standard care. The main goal is …

This study is observing adults with hemophilia A who are taking the medication Jivi to see if long-term use improves their joint health. Researchers will use ultrasound scans to check the condition of participants' joints before starting Jivi and again after two years of treatmen…

This study aims to understand how well the medication Jivi works for people with Hemophilia A in their daily lives. It will follow 371 patients who have been treated before to see how many bleeding episodes they have, monitor side effects, and measure their satisfaction with trea…