|3rd flight of the Dipsea Steps in|
Fibrodysplasia Ossificans Progressiva is a rare and debilitating genetic condition that essentially turns muscle into bone, creating a second skeleton in the body. Currently there is no known cure. There are 800 people in the world affected with FOP - 285 of those people are here in the United States.
Hayden was diagnosed with FOP almost 14 years ago. Since then, the FOP lab at the University of Pennsylvania, has discovered that the ACVR1 gene causes FOP. Armed with that information, the FOP Lab in conjunction with the International FOP Association, have reached out to pharmaceutical companies to explore the possibility of a cure.
|Hayden and his sister Elsa|
Today, we are in Phase 2 drug trails with Clementia Pharmaceuticals for a drug called Palovarotine. The drug is showing much promise. Hayden is currently in the process of enrolling in Clementia's Natural History Study which will help researchers understand the progression of FOP and how an investigational treatment might affect the course of the disease.
FOP is designated as a rare disease by National Organization for Rare Diseases. Few rare diseases have a therapy or cure. Most funds for research for a rare disease come soley from family fundraisers. The FOP lab at the Department of Orthopaedic Surgery at U of Penn has a budget of $1.5 million/year for research. 75% of these funds come from family fundraising and donations. 25% come from institutional support (NIH/NIAMS, Orthopaedic Research & Education Foundation).
|Hayden and his cousins on |
his 16th birthday this year.
To learn more about the International FOP Association go to www.ifopa.org.