A new gene therapy appears to be safe in patients diagnosed with Friedreich ataxia cardiomyopathy, a progressive and fatal inherited cardiac disease, according to a phase 1 clinical trial led by Weill Cornell Medicine researchers. ...
Jun 17, 2026
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Researchers have uncovered a fundamental rule that governs how genes are physically arranged inside the cell nucleus, and how disruptions to that organization can contribute to human disease.
May 12, 2026
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Friedreich's ataxia (FA) is a rare but devastating genetic disorder. Those with the condition are often diagnosed between 5 and 15 years of age and live only into their 30s or 40s. There is no widely approved treatment that ...
Dec 10, 2025
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Broad Institute researchers have developed a way to edit the genetic sequences at the root of Huntington's disease and Friedreich's ataxia.
May 27, 2025
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Many diseases are caused by a missing or defective copy of a single gene. For decades, scientists have been working on gene therapy treatments that could cure such diseases by delivering a new copy of the missing genes to ...
Apr 28, 2025
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Friedreich's ataxia is a rare, inherited disease that causes progressive nervous system damage, impairing balance and coordination and leaving patients unable to walk by early adulthood.
Jun 22, 2023
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Oxidative stress—characterized by elevated levels of unstable molecules called reactive oxygen species—is associated with neurodegeneration and cardiovascular disease. However, until recently it has not been possible to demonstrate ...
Jun 14, 2023
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A multi-disciplinary team of researchers has developed a way to monitor the progression of movement disorders using motion capture technology and AI.
Jan 22, 2023
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Researchers at the University of Miami Miller School of Medicine, McGill University and other institutions have found that a well-concealed genetic variation in the gene FGF14, called a DNA tandem repeat expansion, causes ...
Dec 14, 2022
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