Targeting to BMP9 to restrain flare-up of fibrodysplasia ossificans progressiva

靶向 BMP9 以抑制进行性骨化性纤维发育不良症的爆发

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Abstract

Fibrodysplasia ossificans progressiva (FOP) is a rare genetic disease characterized primarily by the formation of heterotopic bone in connective tissue, such as tendon, muscle, ligament and fascia (Kaplan et al, 2024). The prevalence is approximately one in 2.5 million births, with over 2,500 cases diagnosed globally. Although rare, FOP is a severe and life-limiting condition with no current cure. Due to heterotopic bone formation in various organs, patients experience intense pain, restricted mobility, difficulty with eating and speaking, hearing loss, scoliosis, and impaired pulmonary function. The average lifespan for individuals with FOP is around 40 years. Therefore, studying the pathological mechanism and developing efficient drugs for FOP are highly demanding. In this issue of EMBO Molecular Medicine, Zhao et al, introduce bone morphogenetic protein 9 (BMP9) as a potential therapeutic target for FOP (Zhao et al, 2024).

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