Gene Therapies Could Transform Rare Diseases. Are We Holding Them Back? Gene therapies could transform how we treat rare diseases. But will everyone have access?
They Created a Drug for Susannah. What About Millions of Other Patients? Scientists have made rapid progress in customizing drugs for ultrarare diseases. The hard part now is making such treatments on a large scale.
The Disease Took Zara, Then Sara. Could Ayla Be Saved? Two sisters in one family died from a genetic disorder. Before a third child with the disease was born, doctors hatched a plan to spare her of its effects.