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Anti-TGF-beta therapy promising in osteogenesis imperfecta


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Journal
Journal of Clinical Investigation
Reuters Health - 24/02/2022 - In adults with osteogenesis imperfecta (OI), a single administration of fresolimumab, a pan-anti-TGF-beta neutralizing antibody, improved bone mass and turnover in a phase 1 study. Currently, there is no specific therapy for OI. "We were surprised by the size of the effect on bone mass in the spine of some of the patients with moderate OI," Dr. Brendan Lee of Baylor College of Medicine in Houston told Reuters Health by email. "As this was a phase 1a study that focused on evaluating safety, we did not expect dramatic changes in bone mass. In some of the subjects with moderately severe forms of OI, we saw increases of 6%-8% in bone density at the spine at three-to-six months after a single injection." However, he noted, "This was not seen in all subjects and it will be important to perform follow up repeat studies to determine the range of effect." "We would expect (fresolimumab) to also work in children, but we need to focu...


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