The siRNA drug, sold as Redemplo for familial chylomicronemia syndrome, showed promise in severe hypertriglyceridemia in a study presented at ACC.

At ACC, tonlamarsen reduced angiotensinogen in a trial, but the data raise questions about its duration and how well it can treat hypertension.

Data from the ATOMIC trial in the NEJM offers a practice-changing adjuvant immuno-chemo regimen for colon cancer, and have experts wondering about a chemo-free option.

The recommendation is based on data from a randomized Phase II/III trial and an open-label extension clinical trial of patients with APDS.

After multiple delays, the US regulator has approved Kresladi as the first gene therapy for severe leukocyte adhesion deficiency-I.

The companies submitted data from two Phase II studies showing Ibtrozi's activity in patients who had and hadn't received prior tyrosine kinase inhibitors.

The investigator-initiated study will test whether SRN-101 can safely and effectively drive anti-tumor cytokine production and immune activation.

The appeals panel upheld a portion of appeals raised by Eisai and Eli Lilly over the process NICE used to recommend against ...

NetraMark will analyze biomarker data in patient subgroups to provide deeper insights into which tailored treatments delivered the strongest benefit.

The companies have identified an initial set of genetic biomarkers to predict drug response and will validate the findings in ...

In the randomized, controlled Phase III trial, children will receive either a single injection of ETX101 or be assigned to a sham control group.

The tumor tissue-based real-time PCR kit will be used to identify patients with PIK3CA gene mutations eligible for treatment with PI3K inhibitor.