New Sickle Cell Therapies Will Be Out of Reach Where They Are Needed Most
There is no clear path for African patients to get [...]
Sickle-Cell Treatment Created With Gene Editing Wins U.K. Approval
The first treatment that relies on CRISPR is expected to [...]
A New Sickle Cell Treatment Will Change Lives — but How Many?
A new treatment for sickle cell disease will change lives [...]
FDA Approves Biogen’s Drug for Rare Form of ALS
The drug, which will be sold as Qalsody by the [...]
A Dilemma for Governments: How to Pay for Million-Dollar Therapies
A wave of transformative but hugely expensive treatments is challenging [...]
They Created a Drug for Susannah. What About Millions of Other Patients?
Scientists have made rapid progress in customizing drugs for ultrarare [...]
Alzheimer’s APOE4 Genetic Risk Targeted in Promising Trial
Preliminary results offer hope that gene therapy can protect people [...]
The Disease Took Zara, Then Sara. Could Ayla Be Saved?
Two sisters in one family died from a genetic disorder. [...]
Gene Treatment for Rare Epilepsy Causes Brain Side Effect in 2 Children
The side effect, a buildup of fluid in the brain, [...]
Hospital and Drugmaker Move to Build Vast Database of New Yorkers’ DNA
Patients will be asked if their genetic sequence can be [...]