Magrolimab Plus Azacitidine Versus Placebo Plus Azacitidine in…
A large trial showed adding an immune-boosting antibody to standard MDS treatment didn't improve survival and caused more severe side effects, preventing adoption of a harmful regimen.
- Lab
- Preclinical
- Early trials
- Late trials
- In practice
Landscape of parental postzygotic mutations across >11,000 rare disease trios.
Researchers identified a hidden class of genetic mutations in disease-causing genes that standard DNA testing misses, improving how doctors estimate recurrence risk in rare disease families.
Novel or significantly improved treatmentSwitching to camizestrant at ESR1 mutation emergence before disease…
Blood tests detecting treatment-resistant cancer mutations allowed doctors to switch therapy early, nearly doubling progression-free survival in advanced breast cancer patients.
Novel or significantly improved treatmentEfficacy and safety of dabrafenib plus trametinib in adults with…
This global phase 3, double-blind, placebo-controlled trial randomized 153 previously treated patients with BRAF V600E-positive RAI-refractory differentiated thyroid cancer 2:1 to dabrafenib plus trametinib or placebo, demonstrating a 3.5-fold improvement in median PFS (12.8 vs 3.7 months) and a 57% objective response rate versus 4% with placebo. The consistent benefit and manageable safety profile support this BRAF/MEK inhibitor combination as a new second-line standard for this genomically defined thyroid cancer subtype.
Novel or significantly improved treatmentAn anti-PMEL antibody-drug conjugate with a G(q/1)(1) inhibitor…
A custom-designed antibody drug targeting a specific mutation showed activity in uveal melanoma, a cancer with no approved treatments and extremely poor outcomes.