In a Phase I study, previously treated pediatric SMA patients exhibited reductions in a marker of neurodegeneration after salanersen treatment.

In a Phase I/II clinical trial, TKI-pretreated NSCLC patients given zidesamtinib had an overall response rate of 44 percent.

The funding will support the development of Revolution's RAS(ON) inhibitor daraxonrasib in exchange for tiered royalties on its sales.

UCLA-led investigators say additional research is needed into why guideline-recommended genetic testing remains low among ...

In the NEULARK trial, patients with early-stage LRRK2-driven Parkinson's disease will receive Neuron23's NEU-411 or a placebo.

The funding comes from the Foundation Fighting Blindness' venture philanthropy arm, the Retinal Degeneration Fund.

The agency granted the accelerated approval for Datroway in EGFR-mutant NSCLC based on data from the TROPION-Lung05 and TROPION-Lung01 trials.

For selected applicants, the agency plans to cut review times down to one to two months and provide "tumor board-style" ...

Top-line results from a Phase III trial showed an overall survival benefit for zanzalintinib combo therapy in microsatellite-stable colorectal cancer.

In the growing rare disease precision medicine market, drug companies must avoid funding genetic testing that the Justice ...