All posts by Megha Shree


FDA Approves First Treatment for Fibrodysplasia Ossificans Progressiva

Action The U.S. Food and Drug Administration (FDA) has approved Sohonos (palovarotene) capsules for reduction in the volume of new heterotopic ossification (extra-skeletal bone formation) in adults and children aged 8 years and older for females, and 10 years and older for males with fibrodysplasia ossificans progressiva. Sohonos is the first drug approved for patients with fibrodysplasia […]

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Abnormal Bone

Drug Trial Shows Reduced Abnormal Bone Formation in Those with Fibrodysplasia Ossificans Progressiva

A multisite, international phase 2 trial evaluating the investigational drug garetosmab has shown that it reduced soft-tissue flare-ups significantly and prevented new areas of abnormal bone formation in patients with fibrodysplasia ossificans progressiva (FOP). Kathryn Dahir, MD, professor of Medicine in the Division of Endocrinology and Diabetes, served as the principal investigator at Vanderbilt University […]

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Ongoing Clinical

Ongoing Clinical Trials Offer Hope for People Born with Genetic Mutation Behind Fibrodysplasia Ossificans

BLU-782 is effective in preventing heterotopic ossification after fibular osteotomy. Credit: Science Translational Medicine (2024). DOI: 10.1126/scitranslmed.abp8334 Several teams of medical researchers are currently testing new therapies for slowing or stopping the excess or runaway bone growth associated with fibrodysplasia ossificans progressive (FOP)—a rare bone disease. The team behind one such effort, with members from Blueprint Medicines […]

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ALK2 Inhibitor

An ALK2 Inhibitor, BLU-782, Prevents Heterotopic Ossification in a Mouse Model of Fibrodysplasia Ossificans Progressiva

Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease driven by gain-of-function variants in activin receptor-like kinase 2 (ALK2), the most common variant being ALK2R206H . In FOP, ALK2 variants display increased and dysregulated signaling through the bone morphogenetic protein (BMP) pathway resulting in progressive and permanent replacement of skeletal muscle and connective tissues with […]

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Fibrodysplasia Ossificans Progressiva

Long-Term Use of Interleukin-1 Inhibitors Reduce Flare Activity in Patients with Fibrodysplasia Ossificans Progressiva

Introduction Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disease with an estimated prevalence of 1:1.2–1.5 million [1]. It is among the most catastrophic forms of heterotopic ossification (HO) [2] because of the progressive, irreversible accumulation of heterotopic bone and subsequent loss of mobility and independence. FOP is caused by activating mutations (mostly, the highly […]

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PI3Kα Inhibition Blocks

PI3Kα Inhibition Blocks Osteochondroprogenitor Specification and the Hyper-Inflammatory Response to Prevent Heterotopic Ossification

Introduction Heterotopic ossification (HO) is a disorder characterized by ectopic bone formation at extraskeletal sites, including skeletal muscle and connective tissues. Trauma-induced HO develops as a common post-operative complication after orthopedic surgeries (e.g., hip arthroplasty), blast injuries, deep burns and nervous system injuries (Hwang et al., 2022). In addition to trauma- induced HO, fibrodysplasia ossificans […]

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