Abstract Search

ba0006p138 | (1) | ICCBH2017

Fibrodysplasia ossificans progressiva: baseline characteristics of 101 subjects participating in a global, longitudinal, natural history study

Kaplan Frederick S , Hsiao Edward C , Baujat Genevieve , Brown Matthew A , De Cunto Carmen , Di Rocco Maja , Keen Richard , Al Makkadam Mona , Grogan Donna R , Pignolo Robert J

Objectives: Progressive heterotopic ossification in fibrodysplasia ossificans progressiva (FOP; OMIM #135100) begins in childhood and leads to irreversible restriction of movement, functional impairment, and shortened life-span. Baseline data from an on-going, global, 3-year, natural history study (NHS) describe FOP disease characteristics, and retrospective flare-up history, causes/symptoms, and outcomes.Methods: Data from 101 subjects (recruited from 2...

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ba0007oc27 | (1) | ICCBH2019

Palovarotene inhibits the development of new heterotopic ossification in fibrodysplasia ossificans progressiva (FOP)

Kaplan Frederick , Hsiao Edward C , Baujat Genevieve , Keen Richard , De Cunto Carmen , Di Rocco Maja , Brown Matthew A , Al Mukaddam Mona M , Grogan Donna R , Pignolo Robert J

Objective: FOP is a rare, severely disabling disease characterized by episodic flare-ups and accumulation of heterotopic ossification (HO) leading to restricted movement, physical disability, and early death. Data from two Phase 2 interventional studies and one natural history study (NHS) were used to evaluate whether palovarotene could reduce HO following an FOP flare-up.Methods: HO volume at the flare-up site was determined by CT at baseline and 12 wee...

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Bone Abstracts

Fibrodysplasia ossificans progressiva: baseline characteristics of 101 subjects participating in a global, longitudinal, natural history study

Palovarotene inhibits the development of new heterotopic ossification in fibrodysplasia ossificans progressiva (FOP)

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