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Orthopaedic Institute for Children’s Research May Lead to Treatments for Patients with Rare Bone Disorders
Apr. 04, 2016 09:39AM PST
Genetics InvestingBreakthrough research being funded by the Orthopaedic Institute for Children (OIC) is creating new hope for children afflicted with a rare genetic disorder that turns muscle, tendons and ligaments into bone, thus constraining movement and ultimately leading to loss of mobility. For those afflicted with this deadly disease, fibrodysplasia ossificans progressiva (FOP), median age of …
Breakthrough research being funded by the Orthopaedic Institute for Children (OIC) is creating new hope for children afflicted with a rare genetic disorder that turns muscle, tendons and ligaments into bone, thus constraining movement and ultimately leading to loss of mobility. For those afflicted with this deadly disease, fibrodysplasia ossificans progressiva (FOP), median age of survival is 40 years or potentially less due to delayed diagnosis, trauma and infections.
Karen Lyons, Ph.D. stated:
FOP is the result of a gene mutation that causes non-bone forming cells to transform into bone-producing cells; as a result, bone forms in locations where it should notOur study showed that any therapy that inhibits the activity of the mutated form of the gene would probably be well tolerated in humans.