MeSH tree: C16.320.322
The U.S. Food and Drug Administration has approved the Zycubo (copper histidinate) injection as the first treatment for Menkes disease in pediatric patients. Approval of Zycubo was granted to Sentynl Therapeutics.
Jan 21, 2026
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A Northwestern Medicine study has uncovered a promising new therapeutic approach for a rare genetic brain disorder, according to findings published in Nature Communications.
Jan 15, 2026
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In research published in Developmental Medicine & Child Neurology, investigators have developed a brief, reliable, and valid screening tool to help identify individuals with Duchenne muscular dystrophy (a neuromuscular disorder) ...
Jan 14, 2026
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Until relatively recently, children and young people with life-shortening conditions were not expected to survive into adulthood.
Jan 7, 2026
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Ongoing clinical research at UNC could lead to a first-of-its-kind enzyme replacement therapy for Hunter syndrome, an ultra-rare disorder that causes progressive multisystem disease and neurologic decline.
Jan 5, 2026
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The U.S. Food and Drug Administration has approved Waskyra (etuvetidigene autotemcel) as the first cell-based gene therapy for the treatment of Wiskott-Aldrich syndrome (WAS).
Dec 16, 2025
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Scientists found that messenger RNA (mRNA) molecules that carry genetic instructions to the far reaches of neurons in the brain tend to cluster together mostly because they are abundant, not because they move in coordinated ...
Dec 15, 2025
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For men with hemophilia B, receipt of gene therapy comprising an infusion of etranacogene dezaparvovec results in sustained endogenous factor IX expression and low annualized bleeding rates over five years, according to a ...
Dec 10, 2025
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The United States on Friday said it would add its most serious warning label to a gene therapy treatment for a debilitating muscle-wasting disease, and also limit its approved use.
Nov 15, 2025
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Nancy Brady has been gratified to see the tool she and colleagues pioneered over a decade ago to measure the growth of infants' pre-speech communication skills translated into several languages and referenced in more than ...
Nov 10, 2025
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