Enhancing Early Recognition and Management of FOP: Strategies for the Pediatric Clinician

Target Audience
This activity is intended for pediatricians, pediatric orthopedic surgeons, medical geneticists, genetic counselors, and other healthcare providers who may participate in the assessment and care of individuals with undiagnosed or diagnosed FOP.

Program Overview
Fibrodysplasia ossificans progressiva (FOP) is an inherited autosomal dominant condition that causes progressive heterotopic ossification (HO), which is the disabling conversion of connective tissue and muscle to bone. Current management strategies center around avoiding events that trigger HO, and a growing understanding of disease pathology has led to the development of targeted therapies, which are being evaluated in advanced phases of clinical trials. These investigational therapies may offer proactive treatment options that mitigate the pathology and progression of FOP. However, as an ultra-rare disease, FOP remains under- or misdiagnosed, and healthcare providers may miss distinct clinical characteristics that are highly suggestive of the disorder.

Patients often undergo unnecessary diagnostic and therapeutic interventions that may cause harm and may be precluded from accessing appropriate management. This educational activity seeks to present pediatric healthcare providers with FOP disease background and strategies to facilitate early recognition and diagnosis, as well as review the most recent clinical trial data of emerging targeted treatment strategies.

Learning Objectives
Upon completion of this activity, the learner should be able to:

Identify symptoms and characteristic clinical presentations of FOP to prompt timely diagnosis
Describe appropriate clinical, imaging, and genetic diagnostic strategies to differentially identify FOP
Review current supportive care principles for FOP management
Evaluate the mechanisms of action, clinical efficacy, and safety data on advanced investigational disease-specific therapies for FOP
Assess potential considerations for the use of emerging FOP-specific therapies in practice