Treatment Considerations for the Management of Fibrodysplasia Ossificans Progressiva

The ultimate goal of research on fibrodysplasia ossificans progressiva (FOP) is the development of treatments that will prevent, half, or even reverse the progression of the condition. Despite advances in understanding the pathophysiology of FOP, there are no therapies with scientifically proven benefits for the prevention or treatment of FOP. Insights obtained from the research laboratory and from the treatment of hundreds of patients world wide have allowed us to identify targets for potential therapies. Some of these therapies involve the use of currently available medications, whereas others involve novel therapeutic approaches that are in preclinical testing. In this article, we will review the scientific basis for considering various therapeutic options based on the known pathophysiology of FOP. Because there are presently no proven preventions or treatments for the condition, this article is intended to represent an opinion of the authors that may be helpful to others who face similar situations.