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.

A sponsor will have to demonstrate success with "with several consecutive patients with different bespoke therapies" to ...

The German company developed 177Lu-edotreotide to treat SSTR-positive gastroenteropancreatic neuroendocrine tumors and expects an agency decision in August.

The group recommends considering risk-reducing surgery for those with ovarian cancer risk and enhanced surveillance to manage breast cancer risk.

The firm will use its AI platform to identify biomarkers of response to combination immunotherapy and chemoradiotherapy.

CTX310 showed promising safety and efficacy in a small Phase I trial, according to data presented at the American Heart ...

Investigators who designed a base-editing treatment are planning an "umbrella of umbrellas" trial, while others are developing roadmaps to guide personalized treatments.