Nakai lab receives three NIH grants to develop potential curative treatments for inherited kidney diseases Hiroyuki Nakai, ...

Valentina Ruggeri’s parents were told their little girl was the only person in Australia with an ultra-rare genetic condition – and there was no cure. So they took matters into their own hands. But ...

Evolving biomarker data show early signs of dose-dependent increase in cerebrospinal fluid (CSF) progranulinSerum ...

Newly announced Phase 4 THRIVE trial results showed most patients who previously had an inadequate response to a ...

A Nobel-winning therapy that can activate nerves with the flick of a switch is also being turned into treatments for deafness ...

‘Triple-negative breast cancer’, which accounts for about 20% of all breast cancers, has been difficult to treat because available targeted therapies are limited. A study has reported that ...

Rocket Pharmaceuticals (RCKT) rose in afternoon trading on Thursday after Goldman Sachs upgraded the gene therapy developer to Neutral from Sell, citing the latest updates on RP-A501, its experimental ...

The 1995 US Open winner announced on Instagram that she had to undergo a cell-based gene therapy as part of treatment. He has ...

Editas Medicine (EDIT) traded higher on Thursday after Australian authorities cleared a Phase 1/2 trial for the company’s gene-editing therapy EDIT-401, and Wells Fargo upgraded the biotech to ...

Within hours of being born, Henry Dotson got fussy and refused to eat — a warning sign that led doctors to a terrifying ...

Researchers report encouraging findings from the first clinical cohort of blind people treated with optogenetic therapy ...