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TAZ gene replacement therapy
TAFAZZIN gene replacement therapy
Background
Barth Syndrome (BTHS) is an X-linked, potentially lethal genetic disease that affects about 1 in 0.3 to 0.4 million live births. It is characterized by cardiomyopathy, skeletal myopathy, neutropenia, growth delay, poor feeding, and organic aciduria, with cardiac complications and neutropenia being the...
Published: 3/30/2026
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Updated: 3/10/2025
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Inventor(s): William Pu, Suya Wang
Keywords(s):
Category(s): Technology Classifications > Disease Area > Rare Diseases, Technology Classifications > Invention Type > Therapeutics > Gene Therapy, Technology Classifications > Disease Area > Cardiology
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