To the research team working to save him, KJ Muldoon was first known only as Patient Eta. But within months, KJ’s name — and ...

Now Ollie is making history as the world’s first successful gene therapy patient for this cruel condition. Ten months after ...

A California toddler is the first person in the world to receive gene therapy to treat his devastating disease. Three-year-old Oliver Chu was born with a rare, genetic condition called Hunter syndrome ...

Oliver has an inherited condition called Hunter syndrome, which causes progressive damage to the body and brain.

Three-year-old Ollie Chu, born with the rare and life-limiting Hunter syndrome, is showing improvement after receiving an ...

FDA reports rare neutralizing antibodies, including a pediatric death, in patients treated with Takeda's Adzynma for congenital TTP.

Patna: Once dismissed as a relic of medieval medicine, leech therapy is crawling back into Bihar’s healing scene. What was ...

Lynk received the green light from the FDA to begin clinical trials of its investigational oral therapy LNK01006 for MS and other diseases.

Pirtobrutinib, a non-covalent Bruton tyrosine kinase (BTK) inhibitor, met the primary endpoint for non-inferiority in terms of overall response rate in the first head-to-head comparison with ibrutinib ...

Researchers have identified how a single mutation in the GPX4 enzyme causes neurons to undergo ferroptosis, leading to severe early-onset dementia.

Nobel laureate David Baker’s latest foundation model can design proteins that interact to any biomolecule for broad biotech applications.