Mydicar is an experimental gene therapy that has been studied as a treatment for severe heart failure. It is described as a genetically targeted enzyme replacement therapy designed to raise the level of SERCA2a, a calcium ATPase found in the membrane of the sarcoplasmic reticulum of heart muscle cells. The SERCA2a gene is delivered to the heart by an adeno-associated viral vector, and the use of the alpha-myosin heavy chain gene promoter directs expression only to cardiac muscle cells. According to the source, Mydicar was compared with a placebo in a phase 2 study of 39 advanced heart failure patients, and treated patients showed a 52 percent reduction in the risk of worsening heart failure. It is a research product, not an established treatment, and nothing here is medical advice.
Facts
Classification
Approval StatusInvestigational / Clinical Trial 1 ATC ClassC: Cardiovascular System 1 Connections
Treats
Source Mydicar (Wikipedia)
Sources
1. Mydicar (Wikipedia)
Lead paragraph
Mydicar is a genetically targeted enzyme replacement therapy being studied for use in patients with severe heart failure.
Lead paragraph [approval-status]
Mydicar is being tested in a phase 2 study
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