Palovarotene for Fibrodysplasia Ossificans Progressiva (FOP): Results of a Randomized, Placebo‐Controlled, Double‐Blind Phase 2 Trial
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DOI: 10.1002/jbmr.4655
Abstract: Fibrodysplasia ossificans progressiva (FOP) is an ultra‐rare genetic disorder characterized by progressive heterotopic ossification (HO), often heralded by flare‐ups, leading to reduced movement and life expectancy. This placebo‐controlled, double‐blind trial (NCT02190747) evaluated palovarotene, an orally… read more here.
Keywords: progressiva fop; placebo; ossificans progressiva; week ... See more keywords
Self-reported baseline phenotypes from the International Fibrodysplasia Ossificans Progressiva (FOP) Association Global Registry.
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DOI: 10.1016/j.bone.2020.115274
Abstract: A global, patient-reported registry has been established to characterize the course of disease and track clinical outcomes in patients with fibrodysplasia ossificans progressiva (FOP), an ultra-rare genetic condition of progressive heterotopic ossification (HO) that results… read more here.
Keywords: progressiva fop; fibrodysplasia ossificans; baseline phenotypes; fop association ... See more keywords