Gene therapies could transform how we treat rare diseases. But will everyone have access?
Tag: Rare and Orphan Diseases
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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.
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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.