HEMOPHILIA A

This study tested a single-dose gene therapy called SB-525 for adults with severe hemophilia A. The goal was to see if it was safe and if it could teach the liver to produce its own missing clotting factor (Factor VIII) for years. If successful, this one-time treatment could prov…

This study tested the safety of an extended-release clotting factor injection called BAY94-9027 (Jivi) in children aged 7-12 with severe hemophilia A. The treatment is designed to last longer in the body, potentially requiring fewer injections to prevent and control bleeding epis…

This study tested a new, long-acting version of the missing clotting factor (called BIVV001) in children under 12 with severe hemophilia A. The main goal was to see if it was safe and effective at preventing and treating bleeding episodes over a year. The study involved 74 childr…

This study monitored the safety and effectiveness of Kovaltry, a medication for hemophilia A, during routine medical use. Researchers followed 230 patients, including 50 children under 12, for up to two years to track side effects and how well the drug controlled bleeding episode…

This early-stage study tested a single intravenous infusion of an experimental gene therapy called BAX 888 in adult men with severe hemophilia A. The main goals were to check for side effects and find a safe and effective dose. Researchers hoped the therapy would help the body pr…

This study tested a new scoring system to help doctors personalize treatment plans for people with hemophilia A when switching to a longer-lasting medication called Jivi. The score used factors like a person's bleeding history and activity level to assign one of three dosing sche…

This study tested a preventive treatment called emicizumab in people with mild or moderate hemophilia A who do not have inhibitors. The goal was to see if regular injections could safely reduce the number of bleeding episodes. Researchers measured how often participants bled and …

This study tested a single-dose gene therapy, called valoctocogene roxaparvovec, in 22 adult men with severe hemophilia A. The goal was to see if the therapy could safely help the liver produce its own blood-clotting factor (Factor VIII), reducing or eliminating the need for regu…

This study tested whether a new preventive drug called emicizumab could reduce the number of bleeding episodes in people with hemophilia A. It involved 85 participants, including teens and adults, who were prone to frequent bleeds. They were given either the drug as a weekly or m…

This small, early study aimed to measure how long a new experimental drug (BIVV001) stays active in the body compared to two standard treatments for severe hemophilia A. It involved 13 adult men who had previously been treated for their condition. Researchers gave each participan…

This study collected real-world information about how doctors use the medication emicizumab to prevent bleeding in very young children (ages 0-3) with hemophilia A. Researchers reviewed medical records from 71 children across several treatment centers to understand common dosing …

This study aimed to collect detailed information on how well current standard treatments work for adults with severe hemophilia A or B. Researchers tracked bleeding rates and treatment use in 212 participants for at least six months. This data serves as a crucial baseline to comp…