Even though the treatment options that exist for acute myeloid leukemia (AML) are growing, the clinical outcome of patients is still unfavorable. In AML dysregulation of the tyrosine kinase receptors, including RAS, RAF, MEK and ERK, and of the Aurora kinase family (AURK) exists, are both tied to AML development and progression.

Variants in several subunits of the Mediator protein complex are responsible for MEDopathies, which present variable clinical manifestations and modes of inheritance. Researchers from Université de Paris Cité have investigated the role of MED16, a subunit of the evolutionary-conserved Mediator complex, in MEDopathies.

Atrial septal defect (ASD) is a congenital heart condition where the formation of the connection between the atrial chambers is defective, thus allowing left-to-right shunting and the consequent risk of atrial fibrillation, stroke and heart failure, among others.

Septerna Inc. scientists have reported proof-of-concept data on a novel oral small-molecule thyroid-stimulating hormone receptor (TSHR) antagonist, SP-1351, with potential for the treatment of Graves’ disease (GD), including ophthalmic manifestations.

The therapeutic response of approved KRAS inhibitors is not always durable and patients develop resistance frequently. To enhance this durability, the search for KRAS inhibitors in combination with drugs that target downstream or upstream components of RAS is crucial.

Philochem AG has discovered an ultra-high-affinity ACP3 ligand – OncoACP3 – isolated through the screening of DNA-encoded chemical libraries for the potential treatment of prostate cancer.

It is known that >70% of the genes involved in pathogenic hearing loss are involved in autosomal recessive nonsyndromic hearing loss (ARNSHL), which accounts for 1 case for every 166 individuals in Iran due to the high rate of consanguineous marriage in the country.