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ea0070aep1025 | Hot topics (including COVID-19) | ECE2020

Medications used by individuals with fibrodysplasia ossificans progressiva (FOP): Data from a global natural history study

C. Hsiao Edward , Al Mukaddam Mona , Baujat Geneviève , De Cunto Carmen , Keen Richard , J Pignolo Robert , Harnett Kathleen , Marino Rose , Kaplan Frederick S

Background: FOP is an ultra-rare genetic disorder characterised by episodic progressive heterotopic ossification (HO) and flare-ups, causing cumulative disability and early death. FOP is diagnosed and managed by multiple specialists, including endocrinologists. There are no established disease-modifying therapies to prevent HO in FOP. Treatment guidelines for symptomatic relief of FOP have recently been published by the International Clinical Council on FOP (ICC).1<...

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ea0070aep1019 | Hot topics (including COVID-19) | ECE2020

A global natural history study of Fibrodysplasia Ossificans Progressiva (FOP): 12-month outcomes

Mukaddam Mona Al , Pignolo Robert J. , Baujat Geneviève , Brown Matthew A , De Cunto Carmen , Di Rocco Maja , Hsiao Edward C. , Keen Richard , Le Quan Sang Kim-Hanh , Strahs Andrew , Marino Rose , Kaplan Frederick S

Background: FOP is an ultra-rare, severely disabling genetic disorder characterised by cumulative heterotopic ossification (HO), often preceded by episodic flare-ups, leading to physical disability and early death. Initial misdiagnosis can occur in ~90% of individuals leading to unnecessary, often harmful interventions. FOP is diagnosed and managed by multiple specialists, including endocrinologists.Objective: A prospective, 36-month, global natural...

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Endocrine Abstracts

Medications used by individuals with fibrodysplasia ossificans progressiva (FOP): Data from a global natural history study

A global natural history study of Fibrodysplasia Ossificans Progressiva (FOP): 12-month outcomes

BiosciAbstracts