Shifting Towards Empagliflozin First-Line Therapy in Glycogen Storage…
A diabetes drug given first works better than standard treatments for a rare metabolic disorder, cutting infections and hospitalizations in children.
- Lab
- Preclinical
- Early trials
- Late trials
- In practice
Targeting the nuclear export receptor exportin-1 in acute myeloid leukaemia
Targeting a specific cancer protein with newer drugs shows promise in certain leukemia patients, with tests helping predict who will benefit most.
Near-term implementable findingAumolertinib in non-small cell lung cancer with uncommon EGFR exon 19 deletions
A lung cancer drug works consistently well for patients with a specific genetic variant, offering clearer guidance when detailed genetic testing isn't available.
Standard additionThe association between triglyceride glucose-frailty index and…
Combining blood sugar and muscle-weakness measures predicts heart and metabolic disease risk better than either alone, helping identify vulnerable aging adults.
Underserved or high-risk populationsSurvival and Clinical Progression in Barth Syndrome
The largest study of a rare genetic heart disease reveals early childhood as the critical window for intervention, with transplant access saving lives.