hrp0094ha1 | A Global Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP): 36-Month Outcomes in Participants Aged <25 Years | ESPE2021
Pignolo Robert J.
, Baujat Genevieve
, Brown Matthew A.
, De Cunto Carmen L.
, Hsiao Edward C.
, Keen Richard
, Al Mukaddam Mona
, Le Quan Sang Kim-Hanh
, Marino Rose
, Houchard Aude
, Kaplan Frederick S.
,
Background: FOP is an ultra-rare, severely disabling genetic disorder characterised by progressive heterotopic ossification (HO) following flare-ups. The median age at diagnosis is 5 years, and patients are managed by multiple specialties. No study to date has provided a longitudinal evaluation of FOP. Final data are presented for participants, aged <25 years, enrolled in the first 36-month, prospective, global natural history study of FOP (NCT02322255).</...