MeSH tree: C16.320.400.780
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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Essential tremor (ET) is a common movement disorder affecting about 2% of the American population, and more than 20% of those over 90 years old. Despite its prevalence and decades of study, researchers don't know the precise ...
Dec 5, 2025
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In fatal genetic diseases like Huntington's and spinocerebellar ataxia, proteins develop long stretches of repeating letters that are prone to sticking together like Velcro.
Sep 24, 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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For the first time, an international study has deciphered the genome of the population of Brazil. Published in the journal Science, the research includes the African, Native American, and European ancestries making up this ...
May 15, 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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Ndeye Lam visits the cemetery often, praying and gently touching the seashells laid out across her daughter's gravesite.
Apr 27, 2025
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A team has conducted a trial on the efficacy and safety of L-arginine in treating spinocerebellar ataxia type 6 (SCA6).
Dec 4, 2024
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