It’s highly unusual for an FDA commissioner to discuss drugs with pending applications in the media. It also demonstrates how the agency is raising the bar for firms seeking acc ...

Will Forte, who played MacGruber on Saturday Night Live, and his brother-in-law Doug Modling, who has Huntington's Disease, ...

Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease and sickle cell disease, affect many thousands, others only ...

Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, hemophilia and, recently, Huntington's disease, a fatal neurological ...

"Monogenic" diseases, triggered by mutations in just one gene, may actually be more complex than scientists thought.

Neurogene received FDA Breakthrough Therapy Designation for NGN-401 in Rett Syndrome, driving a >25% share price surge. Click ...

Remarks the FDA commissioner made during a CNBC appearance seemed to stoke investor fears that UniQure's gene therapy for Huntington's won't get approved.

Huntington Local Schools is rolling out new safety protocols after a series of threats that has included seven bomb threats ...

Rare Disease Day is held on the last day of February to raise awareness for rare diseases and address drug development ...

Without naming a specific product, Commissioner Marty Makary referred to an investigational therapy, delivered surgically ...

Charging documents say Marsean Bowles, 19, is charged with felonious assault. He and the victim reportedly met in the parking ...

A panel of physicians, biotech leaders and patient advocates took aim at the FDA during a Senate hearing Thursday on how the ...