Natural history of fibrodysplasia ossificans progressiva: cross-sectional analysis of annotated baseline phenotypes - Pignolo, Robert J.; Baujat, Geneviève; Brown, Matthew A.; De Cunto, Carmen; Di Rocco, Maja; Hsiao, Edward C.; Keen, Richard; Al Mukaddam, Mona; Sang, Kim-Hanh Le Quan; Wilson, Amy; White, Barbara; Grogan, Donna R.; et al. (Orphanet journal of rare diseases, 2019)
Keywords: Adolescent; Adult; Age; Biomarkers; Birth defects; Blood; Child; Child, Preschool; Clinical trial endpoints; Clinical trials; Clinical Trials as Topic; Congenital defects; Cross-sectional analysis; Cross-Sectional Studies; Cytokines; Development and progression; Disease Progression; Dysplasia; Fascia; Female; Fibrodysplasia Ossificans Progressiva; Genetic aspects; Genetic disorders; Health aspects; Heterotopic ossification; Humans; Identification and classification; Information systems; Laboratories; Ligaments; Male; Medical imaging; Medical research; Middle Aged; Muscles; Mutation; Myositis ossificans; Myositis Ossificans - pathology; Natural history; Objectives; Ossification (ectopic); Ossification, Heterotopic - pathology; Patients; Phenotypes; Phosphatase; Product development; Proteins; Rare diseases; Regression analysis; Statistical analysis; Surveys and Questionnaires; Tendons; Tomography; Urine; Young Adult; United States--US; Heterotopic ossification; Clinical trial endpoints; Cross-sectional analysis; Fibrodysplasia Ossificans Progressiva; Natural history; Disease progression