Author Archives: Forest Ray PhD

A first patient in the U.S. has enrolled in a multinational study to collect real-world data on different approaches in managing hemophilia A in people with inhibitors, with an aim of understanding how treatment decisions affect patients. The investigator-led MOTIVATE study (NCT04023019; EudraCT No. 2019-003427-38) is recruiting…

Mobility problems, bleeding, pain, and uncertainly in daily life are the biggest challenges people with hemophilia are facing, according to a survey from six central European countries. Additionally, while most respondents, including caregivers, felt well informed on hemophilia, they continue to seek further education on the condition and new…

Healthcare costs can be 25 times higher for hemophilia B patients than for individuals who do not have a bleeding disorder, according to a recent study. These findings reflect both direct costs of hemophilia treatment and a greater need for other healthcare resources, researchers noted. The study, “…

Both swimming and hydrotherapy can improve functional capacity in hemophilia patients, according to data from a recent study. Of those two types of aquatic exercises, however, hydrotherapy was associated with the most significant functional improvements in these patients. Those findings were reported in the study, “Aquatic…

Exercises to control posture could improve joint health in children with hemophilia and should form part of regular rehabilitation programs, a study suggests. The balance disturbances uncovered in the study could provide early signs of musculoskeletal disorders that could be prevented or reduced through prompt diagnosis and therapy, researchers…

Immunoadsorption, or the selective removal of antibodies from the blood, might prove useful as an add-on treatment for patients with acquired hemophilia, according to a recent study. If confirmed, this finding could lead to better treatment for life-threatening bleeds, delayed treatment responses, and adverse responses to immunosuppressive therapy among…

Although hemophilia treatments are generally effective, women and girls with this disease have specific medical challenges, such as excessive menstrual bleeding, that must be addressed for better clinical care. A study evaluated the symptoms, bleeding management, and clinical outcomes related to menstruation, childbirth, dental procedures, surgeries, and other bleeding events among female…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Two potential gene therapies — AMT-060 and AMT-061 (etranacogene dezaparvovec) — lead to safe and long-term increases in factor IX (FIX) activity, as well as fewer bleeds among patients with hemophilia B, according to the therapies’ maker, uniQure. The company announced in a press release that…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to marzeptacog alfa activated (MarzAA), a lab-made version of clotting factor VIIa for the subcutaneous (under-the-skin) treatment of episodic bleeds in hemophilia A and B patients with inhibitors. MarzAA, developed by Catalyst Biosciences,…