New Gene-Editing Strategy Could Help Development of Treatments for Rare Diseases
Instead of requiring personalized gene edits for each patient, the [...]
These Patients Got the Cure. Then It Went Away.
Gene therapies for rare diseases are frequently developed then discarded [...]
Top F.D.A. Official Resigns Under Pressure
After turning down several new drugs and restricting use of [...]
Drugmakers Notch a $5 Billion Win in Republicans’ Policy Bill
More medicines will be spared from Medicare price negotiations, a [...]
Los médicos le dijeron que iba a morir, pero la IA le salvó la vida
Los científicos están utilizando el aprendizaje automático para encontrar nuevos [...]
Drugs Have Uses We Can’t Imagine. He’s Using A.I. to Find Them.
Scientists are using machine learning to find new treatments among [...]
Can Marty Makary Handle the F.D.A’.s Central Dilemma?
All eyes on Dr. Marty Makary.
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. [...]