The disease is caused by mutations in a single gene. Can gene editing fix the problem?

Cancer treatments from Novartis and Gilead earn approval from EU regulators, but a first pass by the UK’s state-funded health service finds CAR T is too expensive. 

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

The agency proposes ceding its scrutiny of these studies to the FDA.

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.