If approved, Ojemda® (tovorafenib) is expected to be the first and only targeted medicine in European Union for children with relapsed or refractory BRAF-altered pediatric low-grade glioma, ...

Pasithea Therapeutics Corp. ( KTTA) Oppenheimer 36th Annual Healthcare Life Sciences Conference February 26, 2026 4:00 PM EST ...

Initial Phase 1 data of zovegalisib in PIK3CA-driven vascular anomalies expected to be announced in first half of 2026 Breast cancer triplet data and frontline Phase 3 plans expected to be announced ...

Orsini, a leader in rare disease pharmacy solutions, has been selected by Genetix Biotherapeutics Inc. as a specialty ...

Being invited to present research at an international academic conference is an honor for any seasoned professional. But for ...

Gene therapies have the potential to cure some diseases, but they are extraordinarily expensive. Location can also be a big ...

Crispr’s ability to cut genetic code like scissors has just started to turn into medicines. Now, gene editing pioneer ...

When the Chernobyl power plant explosion scattered ionizing radiation all over Europe, the damage it dealt lasted much longer than the initial blast. Researchers sequenced the genomes of Chernobyl ...

Scientists have created a complete map showing how hundreds of possible mutations in a key cancer gene influence tumor growth. The study focused on CTNNB1, a gene that produces the protein β-catenin, ...

An international team of scientists headed by researchers at the University of Edinburgh, has created a complete map showing how hundreds of possible mutations in a key cancer gene, CTNNB1, influence ...

To share your story, or find out how to get involved with CNBC Cures, email CNBC.cures@cnbc.com Susannah Rosen was diagnosed with a KIF1A gene mutation at the age of 2½. Patients diagnosed with severe ...