Researchers have discovered a new way to increase a key brain protein damaged in Rett syndrome, a rare genetic disorder that affects thousands of children worldwide. Early studies in mice and ...

March 6 (Reuters) - The head of the U.S. Food and Drug Administration's vaccines and biologics unit, Dr. Vinay Prasad, will leave the agency at the end of April, a spokesperson for the U.S. Department ...

The FDA’s dispute with biotech firm uniQure over Huntington’s gene therapy highlights growing tensions between innovation, ...

Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced presentations on SGT-003, its ...

A gene borrowed from the naked mole rat, an animal famous for extraordinary longevity and disease resistance, helped mice live a little longer and stay healthier, pointing to a surprising biological ...

A lawmaker compared getting the vaccine with using meth. The Idaho Medical Association said his bill would infringe on medical freedoms.

Subjecting participants to invasive procedures without the prospect of therapeutic benefit is unjustifiable,” says the CEO of ...

A 19-year-old Canadian man becomes the first human cured through prime gene editing after doctors corrected a rare genetic ...

In a strikingly blunt briefing, a senior FDA official accused uniQure of pushing “distorted” data to mask a “failed” therapy ...

Agency officials promise fast reviews of new treatments while vowing they will not be a “rubber stamp” for the industry. But ...

The back-and-forth between the FDA and gene therapy developer uniQure is getting messier, with an agency spokesperson now denying the biotech’s claims about a sham trial request for the rare ...

The FDA has granted Fast Track designation to AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy.