People sometimes asked Tiffany Fransen if she was drunk. She wishes more people had heard of her rare disorder.

A new CRISPR approach can control genes without cutting DNA, opening a safer path for treating genetic diseases. A newly ...

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

The first few months of life are a very important period in terms of human development, and at this point, many of the most ...

Three start-ups are aiming to create gene-edited babies. Columnist Michael Le Page has no doubt that editing our offspring ...

Ultragenyx Pharmaceutical ( ($RARE) ) just unveiled an update. On December 30, 2025, Ultragenyx Pharmaceutical announced it had completed the ...

This year saw notable progress in head and neck cancers, Huntington's disease, personalized genetic therapy, and heart ...

A recent study finds that certain psychiatric disorders have considerable genetic overlap. But these genes aren’t the whole ...

The parents of 7-week-old Dhruvan Gopi were warned he might not survive the night as specialists converged around his crib at ...

Ultragenyx Pharmaceutical said on Monday that late-stage studies showed its drug for a type of genetic bone disease failed to ...

Shaun Carmichael has polycystic kidney disease. He needs a transplant, and his wife is a match to donate. CIRCLEVILLE, Ohio — ...

Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced that it has completed the rolling submission of its Biologics ...