Severe Malignant Osteopetrosis Awareness
July 28, 2015
We’ve all heard of osteoporosis – a common disease as we get older that results in bones that are not dense enough. But have you ever heard of severe malignant osteopetrosis? Unless you’re one of only 200 families in the entire country dealing with this devastating diagnosis… you probably haven’t. But severe malignant osteopetrosis is a very rare bone disease. It afflicts babies and young children, its symptoms are devastating, and the financial burden of treatment options weighs heavily on the families it affects. Thanks to physicians like Dr. Paul Orchard, one of the experts in the U.S. in the use of bone marrow stem cell transplants needed by malignant osteopetrosis patients, life expectancy for these young patients is remarkably improved. And thanks to patient advocates like Paul and Barbara Wersten, who fought for the life of their baby Ryan, support organizations like the Ryan Wersten MIOP Foundation are available to patient families. Currently there is also an FDA-approved treatment called ACTIMMUNE® (Interferon Gamma-1b), marketed by Horizon Pharma Inc., helps delay time to disease progression for patients with SMO. Because of these efforts, and ongoing research families dealing with SMO have more hope today.
On this episode, Beth Troutman tells her story; Christina Nicholson embarks on an adventure to Orlando with her family; and former football player Marc Bouniconti shares his journey.
Three decades later, Marc Buoniconti, a quadriplegic, visits The Balancing Act to talk about his life, his mission, and his goal to one day walk again.
The Balancing Act's host Christina Nicholson and her family are embarking on a fun-filled vacation to Orlando, Fla., also known as the "theme park capital of the world."