A Boston Children’s Hospital trial of a repurposed experimental cancer drug has given parents, physicians, and scientists their first tantalizing evidence that it is possible to affect the course of a rare, fatal disease that causes children to age prematurely and die before reaching adulthood.
The disease, called progeria, affects only about 250 children in the world, but it is known to every medical student because of its heartbreaking trajectory and striking visual presentation, with children that almost seem to shrink into old age just as they are growing up. Children with the disease are far smaller than their peers, lose their hair, have small faces compared with their head size, and die of heart attacks or strokes in their teens.
Comments
I wish research and solutions for Progeria would come faster; I can't believe it's been over 30 years since Aaron Kushner died and Rabbi Harold Kushner wrote his seminal work: ' When Bad Things Happen To Good People'. It's so sad that Orphan Diseaes are lacking funds and support from pharmaceutical companies.
I hope this news continues to be promising.