Rare Childhood Aging Disorder Has New Treatment Hope
A drug traditionally used to prevent the rejection of organ transplants is giving new hope to parents of children with progeria, a rare genetic disorder that causes premature aging and death usually before age 12. Scientists at Children's Hospital and the National Institutes of Health are currently planning a large-scale clinical trial to see whether the drug rapamycin, or a similar medication, might help remove the damaging protein progerin from patients' cells.
According to a report in The Boston Globe:
If successful, the clinical trial could offer hope to those afflicted with the rare disease, which affects an estimated 1 in 4-8 million newborns. It might also shed light on ways to repurpose existing drugs to fight other aging and neurological diseases like Parkinson's.