Fibrodysplasia Ossificans Progressiva Research This is an RSS file. You can use it to subscribe to this data in your favourite RSS reader or to display this data on your own website or blog.

POSA426 Assessment of the Economic Impact of Fibrodysplasia Ossificans Progressiva on Patients and their Families Using an International Burden of Illness Survey
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, severely disabling genetic disorder. Despite recent progress in understanding the clinical characteristics and progression of FOP, a comprehensive assessment of the economic impact of this ultra-rare disease on patients and their families, including direct and indirect medical costs, has not yet been conducted. Here, we describe the economic analysis of the first international, cross-sectional FOP burden of illness online survey (NCT04665323), created in collaboration with FOP community advisors, designed to investigate the economic impact of FOP on patients and...
Source: Value in Health - January 1, 2022 Category: International Medicine & Public Health Authors: M Al Mukaddam, KS Toder, M Davis, K Croskery, AS Grandoulier, J Whalen, FS Kaplan Source Type: research

POSC373 Use of Aids, Assistive Devices and Adaptations by Individuals with Fibrodysplasia Ossificans Progressiva (FOP): 36-Month Results from a Global Natural History Study
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder of progressive heterotopic ossification, causing cumulative disability. Most individuals with FOP become immobilized by the third decade of life, requiring lifelong assistance with activities of daily living. Here, we characterize the use of aids, assistive devices and adaptations (AADAs) as a surrogate measure of FOP progression in individuals with FOP enrolled in a 36-month, global natural history study (NHS; NCT02322255). (Source: Value in Health)
Source: Value in Health - January 1, 2022 Category: International Medicine & Public Health Authors: M Al Mukaddam, G Baujat, A Houchard, EC Hsiao, R Keen, R Marino, RJ Pignolo, FS Kaplan Source Type: research

POSC309 A Pragmatic Patient-Finding Methodology in the Ultra-Rare Disease Fibrodysplasia Ossificans Progressiva (FOP)
For ultra-rare diseases, there are often limited data on the epidemiology, disease characteristics, management practices and geographical location of care. Although robust studies involving large numbers of patients are usually challenging due to the rarity of these conditions, articles reporting patient-level data identified through pragmatic literature reviews (PLRs) may provide valuable insights. (Source: Value in Health)
Source: Value in Health - January 1, 2022 Category: International Medicine & Public Health Authors: K Croskery, A Majdi, K Hanman, E Warnants Source Type: research

Plasma Soluble Biomarkers for Fibrodysplasia Ossificans Progressiva (FOP) Reflect Acute and Chronic Inflammatory States
This article is protected by copyright. All rights reserved. (Source: Journal of Bone and Mineral Research)
Source: Journal of Bone and Mineral Research - December 26, 2021 Category: Orthopaedics Authors: Robert J. Pignolo, Ruth McCarrick ‐Walmsley, Haitao Wang, Shirley Qiu, Jeffrey Hunter, Sharon Barr, Kevin He, Hui Zhang, Frederick S. Kaplan Tags: Original Article Source Type: research

Fibrodysplasia Ossificans Progressiva: What Have We Achieved and Where Are We Now? Follow-up to the 2015 Lorentz Workshop
Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare progressive genetic disease effecting one in a million individuals. During their life, patients with FOP progressively develop bone in the soft tissues resulting in increasing immobility and early death. A mutation in the ACVR1 gene was identified as the causative mutation of FOP in 2006. After this, the pathophysiology of FOP has been further elucidated through the efforts of research groups worldwide. In 2015, a workshop was held to gather these groups and discuss the new challenges in FOP research. Here we present an overview and update on these topics. (Sourc...
Source: Frontiers in Endocrinology - November 10, 2021 Category: Endocrinology Source Type: research