When Michael Pirovolakis received an individualized gene therapy in a single-patient clinical trial at The Hospital for Sick Children (SickKids) in March 2022, the course of his condition was dramatically altered.
Jun 28, 2024
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Drug development is typically slow: the pipeline from basic research discoveries that provide the basis for a new drug to clinical trials to production of a widely available medicine can take decades. But decades can feel ...
Jun 27, 2024
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Researchers at the Pacific Northwest Research Institute (PNRI) and collaborating institutions have made a discovery that could significantly advance our understanding of genomic disorders. Their latest study, published in ...
Jun 21, 2024
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Researchers at the Icahn School of Medicine at Mount Sinai and others have identified a neurodevelopmental disorder, caused by mutations in a single gene, that affects tens of thousands of people worldwide. The work, published ...
May 31, 2024
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Scientists have found the genetic root of a disorder that causes intellectual disability, which they estimate affects as many as one in 20,000 young people. And they hope their discovery leads to a new diagnosis that can ...
Jun 2, 2024
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Using an advanced artificial intelligence tool, researchers at the Icahn School of Medicine at Mount Sinai have identified rare coding variants in 17 genes that shed light on the molecular basis of coronary artery disease ...
Jun 11, 2024
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Foam mixed with medications is already used to treat conditions such as varicose veins, hemorrhoids, wounds on the skin and even hair loss. Now, Fred Hutchinson Cancer Center scientists have found that foam might also be ...
May 29, 2024
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Scientists have discovered a new piece of the puzzle in understanding why some people are more susceptible to Alzheimer's disease. The research has been published in the journal Nature Communications.
Jun 21, 2024
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Researchers have presented the latest findings from a clinical trial aimed at discovering a cure for sickle cell disease, a painful genetic blood disorder with limited treatment options.
Jun 15, 2024
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Researchers at the University of Kentucky Sanders-Brown Center on Aging are working to develop a pre-symptomatic disease diagnostic tool for Alzheimer's disease.
Jun 6, 2024
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