FDA clears first gene therapy for rare disease; Roche’s breast cancer pill win; Women increasingly turn to GLP-1s.

A cutting-edge therapy using base-edited immune cells is offering a major breakthrough for patients with one of the toughest ...

An experimental gene therapy treatment created at UCLA gave a "bubble girl" born with a rare genetic disorder a new life.

From rare diseases to chronic conditions, epigenetic editing unlocks new ways to control gene activity without altering the genetic code.

An $11 million gift was given in the fight against rare genetic diseases.

Chicago's Lurie's Children's Hospital will expand care for patients living with rare genetic diseases and conditions thanks ...

A small group of patients with an otherwise incurable form of T‑cell leukemia have seen their cancer driven into remission by ...

The FDA on Tuesday approved etuvetidigene autotemcel (Waskyra) as the first cell-based gene therapy for patients 6 months and ...

For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five ...

The rate of severe infections decreased from 2 infections to 0.2 infections per PYO after treatment with etuvetidigene autotemcel.

A small group of patients with an otherwise incurable form of T‑ cell leukaemia have seen their cancer driven into remission ...

Don and Anne Edwards have donated $11 million to Ann & Robert H. Lurie Children's Hospital to establish what will be the ...