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
Drug ClassCardiovascular gene therapy (AAV1/SERCA2a) 2 Mechanism of ActionGene therapy that delivers the gene for the SERCA2a enzyme, which is deficient in heart failure 2 Classification
Approval StatusInvestigational / Clinical Trial 1 ATC ClassC: Cardiovascular System 1 Connections
Long-Form Articles
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
View the Source2. Celladon's Mydicar Fails Phase IIb Trial (GEN)
Body, description of Mydicar
Mydicar is designed to work via infusion of the gene for the SERCA2a enzyme, which is deficient in heart failure
Body, first reference to the product
the cardiovascular gene therapy agent Mydicar
Body, CUPID2 result
did not meet its primary and secondary endpoints in the Phase IIb CUPID2 trial for systolic heart failure.
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