The firm is developing the PHOX2B-targeted cell therapy, which has shown promise in preclinical xenograft models, with CHOP researchers.

The firm will stop shipping the gene therapy for these patients until a new immunosuppressive regimen is approved for managing acute liver failure.

Studies doing genome-wide testing can easily ID patients with naturally low neutrophil counts, but nobody's looking at it, ...

Medical ethicists are bringing attention to an early chapter of precision medicine history, an often overlooked aspect of an ...

Trump administration health officials assured drugmakers and researchers at a roundtable hosted by the FDA that they want to ...

Researchers are trying to better understand how co-mutations and PD-L1 expression impact response to single-agent KRAS G12C ...

The firm will use the funds to further develop its lead genetic medicine in Stargardt disease and advance its gene therapy pipeline in other indications.

The drug is already approved for KMT2A-rearranged AML, and Syndax is hoping for approval in NPM1-mutated disease by the end of the year.

Only 6 percent of patients carried a concerning DPYD variant, but nearly two-thirds of them were later hospitalized after ...

A combination of Enhertu and Perjeta nearly doubled progression-free survival compared to standard therapy in data presented ...

As with the drug's 2023 approval in castration-resistant tumors, it's not clear if it works comparably in all homologous ...

The company plans to use some of the funds to support development of a kinase inhibitor in Phase I testing for BCR-ABL fusion-positive chronic myeloid leukemia.