HEMOPHILIA B

This study follows 9 adults with severe or moderately severe hemophilia B who received a one-time gene therapy (AAV5-hFIX) to help their bodies produce clotting factor IX. Researchers are checking if the treatment remains safe and effective for up to 10 years after the initial do…

This study tested a new gene therapy for adults with severe hemophilia B, a bleeding disorder caused by a faulty gene. The treatment uses a harmless virus to deliver a working copy of the factor IX gene, aiming to reduce bleeding episodes. Fourteen participants received one of fo…

This phase 3 trial tested a one-time gene therapy called AMT-061 in 67 men with severe or moderately severe hemophilia B. The therapy uses a harmless virus to deliver a working copy of the gene for clotting factor IX, so the body can produce its own. The main goal was to see if i…

This study followed 151 people with hemophilia B who were already using or starting Alprolix, a long-lasting clotting factor. Over 24 months, researchers tracked how often bleeding occurred, how often the drug was injected, and how much was used. The goal was to see how well Alpr…

This study tested whether a 6-month adapted physical activity program could improve functional capacity in people with hemophilia. Fourteen participants did exercises twice a week to improve joint mobility, strength, and balance. The main goal was to see if their walking distance…

This study looked at whether a special low-intensity exercise program, using blood flow restriction cuffs, is safe and helpful for people with moderate or severe hemophilia. Twenty adults completed 8 weeks of arm and leg exercises. Researchers measured changes in muscle function,…

This study looked at whether manual therapy (hands-on treatment) and structured exercises could improve balance and walking ability in adults with hemophilia. Thirty-five adults with moderate or severe hemophilia took part. Half received 12 physiotherapy sessions over 6 weeks, wh…

This study looked at bone density in 448 men with hemophilia and healthy volunteers to see how common bone loss is. Participants had a bone density scan and some gave blood samples. The goal was to understand if hemophilia itself, not just lack of activity, leads to weaker bones.

This study looked at why people with hemophilia and damaged knee or ankle joints feel ongoing pain. Researchers measured pain sensitivity, fear of movement, and anxiety in 146 adults. The goal was to better understand central sensitization—a condition where the nervous system sta…

This study measured static and dynamic balance in 41 adults with hemophilia and 40 healthy volunteers using a special platform. Researchers also looked at how joint health, physical activity, and fear of movement affect balance. The goal is to understand why balance may be worse …

This study interviewed 150 people with hemophilia B, their caregivers, and healthcare workers from around the world to learn what they know and how they feel about gene therapy. The goal is to create better educational materials and improve the consent process, especially in coun…

This study gathered information on how often adults with moderate to severe hemophilia A or B experience bleeding while on their usual treatment. The data will be used as a baseline for future gene therapy studies. About 212 participants took part, and no experimental treatment w…