BACKGROUND: Genetic variants in components or regulators of the RAS-MAPK signaling pathway are causative for severe and early-onset hypertrophic cardiomyopathy (HCM) in patients with Noonan syndrome ...
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The future of ATTR-CM treatment

Although there is no cure for ATTR-CM, the U.S. Food and Drug Administration has approved three medications for treatment, with other promising medications in the pipeline.
A new case-control study published in JAMA Cardiology demonstrates that molecular imaging using gallium-68–labeled fibroblast ...
End-stage heart disease can leave patients breathless and fatigued despite treatment. Experts explain dilated cardiomyopathy, warning signs, and when a heart transplant becomes necessary, offering a ...
An investigational medicine that addresses the genetic root cause of BAG3 DCM, AFTX-201 is being evaluated as a treatment for BAG3 DCM in the UPBEAT trial in the U.S. and Canada AFTX-201 is designed ...
Mavacamten, the first-in-class cardiac myosin inhibitor approved for treatment of symptomatic adults with obstructive HCM, is efficacious for adolescents.
Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies initially for devastating ...
Nationally, there has been a rise in the number of children and adolescents with congenital and acquired heart disease ...
The expanded program brings together specialists in cardiology, electrophysiology, advanced imaging, heart failure care, and ...
Adolescent patients with obstructive hypertrophic cardiomyopathy (HCM) who received the drug mavacamten saw a significant improvement in left ventricular outflow tract (LVOT) gradient, a measure of ...
Targeting immunometabolic pathways with an investigational glucokinase activator has shown potential for transforming the treatment of diabetic cardiomyopathy beyond glycaemic control in type 2 ...
In a dizzying span of seven months in 2022, Bristol Myers Squibb gained FDA approval for three new products, touting each with the potential to achieve $4 billion in peak sales. While multiple myeloma ...