Current challenges and opportunities in the care of patients with fibrodysplasia ossificans progressiva (FOP): an international, multi-stakeholder perspective.

ABSTRACT:

Background

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, disabling genetic disorder characterized by congenital malformations of the great toes and progressive heterotopic ossification of soft and connective tissues. Assiduous attention to the unmet needs of this patient community is crucial to prevent potential iatrogenic harm and optimize care for individuals with FOP.

Objective

To gather international expert opinion and real-world experience on the key challenges for individuals with FOP and their families, highlight critical gaps in care, communication, and research, and provide recommendations for improvement.

Methods

An international group of expert clinicians, patients and patient advocates, caregivers and representatives from the international

SUBMITTER: Pignolo RJ 

PROVIDER: S-EPMC9014788 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature