FDA, NIH Announce Bespoke Gene Therapy Consortium to Focus on Rare Diseases
More than 30 million people battle rare diseases in the U.S., according to estimates by the FDA.
Baby With Fatal Condition Gets World's Most Expensive Drug
Edward Willis-Hall received the gene therapy drug, Zolgensma, at Sheffield Children's Hospital in England.
Babies with Bubble Boy Disease Cured in World's First
David Vetter, known as "bubble boy," who died in 1984, is perhaps the most famous sufferer of severe combined immunodeficiency.
Blind Boy Might Be Able to See Again After Surgery
"He is dying to see a real rainbow," his mother said.
Gene Therapy Might Prevent Hearing Loss. Should We Try?
Gene therapies are probably still a decade away from being used routinely to treat genetic causes of hearing loss in humans.
First-Ever Gene Therapy Approved Will Treat Blindness
The price likely won't be announced until January, according to a spokesperson.
Hemophilia Might Be Curable With Gene Therapy
The next phase of clinical trials for this remarkable gene therapy may be coming to the U.S. soon.
The Top Medical Stories Of The Year For 2017
From CRISPR bacon to Donald Trump's opioid war declaration, these are the top health stories of 2017.
Latest Gene Therapy Signals Start of a New Medical Era
But these breakthroughs are not going to come cheap.
How Does Gene Therapy for Blindness Work?
The one-time treatment could restore eyesight to thousands of people.
Betting Big on Gene Therapy for the Brain
The decades-old treatment that was until recently thought too dangerous to work could be our only hope.