Investigators at Boston institutions believe the regimen could target immunosuppressive lipid macrophages and improve ...

The agency is cautioning patients about the risk of serious, possibly fatal, liver injuries and has limited the gene therapy's use to ambulatory patients.

The agency reviewed data from a Phase II study, in which around 21 percent of patients had a complete response to the menin inhibitor.

Studies of germline genetic testing programs provide a snapshot of challenges at academic and community practices at the NSGC annual meeting.

NEW YORK – Fondazione Telethon said Friday that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for its gene therapy ...

The committee reviewed data from studies showing that the antisense oligonucleotide reduced the monthly mean rate of angioedema attacks.