Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic ...

While a viral Instagram reel links sudden gym deaths to a genetic heart condition, find out what experts say and why more ...

Trametinib, a mitogen-activated protein kinase (MEK) inhibitor, reduces mortality and morbidity in children with severe ...

Trametinib, a mitogen-activated protein kinase (MEK) inhibitor, reduces mortality and morbidity in children with severe hypertrophic cardiomyopathy (HCM) caused by pathogenic variants in the RAS/MAPK ...

Tenaya plans to present data from its pediatric non-interventional natural history study, known as MyClimb, in the second half of 2025. The MyClimb study was initiated in 2021 to characterize the ...