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.
Walkasins® is a newly available external lower limb sensory prosthesis designed to replace lost nerve function in patients with peripheral neuropathy.
The EB Research Partnership is the largest global organization dedicated to funding research to treat and cure Epidermolysis Bullosa or EB.
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