Epidiolex, a cannabis-derived medicine for epilepsy, will prompt federal regulatory changes that could crack open access to study the plant.

Alnylam’s patisiran interferes with the production of a mutated protein present in people with hereditary transthyretin amyloidosis.

The promise of immunotherapy is real. We now need to figure out how to maximize the number of patients the approach benefits.

Biotechs have developed enzyme replacements and genetically modified probiotics to treat patients with the rare metabolic disorder.

Women with metastatic cancer who were treated with a checkpoint inhibitor had a smaller benefit than did men.

Lucemyra is the first non-opioid drug to be approved in the U.S. specifically for this purpose.

As the first personalized cell and gene therapies are approved from small clinical trials, researchers propose the creation of publicly accessible databases to pull together real-world results.  

In an in vitro study, the compound completely blocked the replication of rhinoviruses.

The scientists involved have hired lawyers to fight the conclusions of a recent investigation into some studies of Diovan in Japan.