F.D.A. Faces Upset Over Denials of New Drugs
Agency officials promise fast reviews of new treatments while vowing [...]
New Gene-Editing Strategy Could Help Development of Treatments for Rare Diseases
Instead of requiring personalized gene edits for each patient, the [...]
F.D.A. Sharply Limits Use of Drug Linked to Two Teen Deaths
A gene therapy for Duchenne muscular dystrophy caused complications to [...]
These Patients Got the Cure. Then It Went Away.
Gene therapies for rare diseases are frequently developed then discarded [...]
Sarepta Refuses FDA Request to Stop Shipping Muscular Dystrophy Drug Elevidys
The regulator had asked Sarepta Therapeutics to halt all shipments [...]
Scientists May Be Able to Make Grapefruits Compatible With Medications They Currently Interfere With
Scientists have identified a gene that causes production of a [...]
First Day of a ‘New Life’ for a Boy With Sickle Cell
Kendric Cromer, 12, is among the first patients to be [...]
F.D.A. Approves 2 Sickle Cell Treatments, One Using CRISPR Gene Editing
People with the genetic disease have new opportunities to eliminate [...]
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 [...]