Alzheimer’s disease has long been a diagnosis that arrives only after memory begins to fray, by which time brain damage is ...

Could one genetic mutation be sufficient to bring on complex psychopathology? For decades, dominant models of mental ...

An Illinois family pushed for both boys to receive the potentially life changing medical treatment after one was approved and ...

An experimental gene therapy seems to slow the progression of Huntington’s disease by about 75 per cent, and researchers are ...

It was the best of times, it was the worst of times for research in 2025. | It was the best of times and it was the worst of times for research in 2025. Medical breakthroughs and stunning discoveries ...

A push to reshore some drug production and progress in advanced manufacturing technologies have been prominent trends this year, industry leaders say.

The Majewski family remains hopeful for their four-year-old son Max, who has a rare neurological disorder, as they seek ...

Now, Musunuru and colleagues are preparing a master clinical protocol to study a CRISPR therapy that's mostly the same as the one they used to treat baby KJ, except they plan to swap out the gene ...

Amid news of the splashy $4.8 billion Amicus buy, BioMarin has slipped in the discontinuation of a genetic liver disease ...

X-linked retinoschisis gene replacement therapy, aiming to improve retinal architecture and visual function in young male ...

The region helped invent cell and gene therapy. Now, there’s an opportunity to reengineer it so more people can actually access it, a life sciences leader writes.

A small group of volunteers will receive multiple injections of the experimental treatments next month, says Unlimited Bio.