Gene Therapies Could Transform Rare Diseases. Are We Holding Them Back?
Gene therapies could transform how we treat rare diseases. But [...]
They Created a Drug for Susannah. What About Millions of Other Patients?
Scientists have made rapid progress in customizing drugs for ultrarare [...]
The Disease Took Zara, Then Sara. Could Ayla Be Saved?
Two sisters in one family died from a genetic disorder. [...]