The NIH’s All of Us Research Program has become the world’s largest integrated genomic and electronic health record database. Its June 30 data release includes ...
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The C9orf72 hexanucleotide repeat expansion is the most common genetic cause of both ALS and frontotemporal dementia (FTD). Carriers face roughly a 50% chance o...
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When a gene therapy sponsor pauses a clinical program, the operational machinery responds within hours. The medical monitor convenes a safety review. Regulatory...
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When Trace Neuroscience announced on June 22 that first patients had been dosed in the LAUNCH ALS trial, it felt like the right moment to revisit our original p...
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On June 23, 2026, the US Department of Health and Human Services launched Operation Trailblazer, a cross-agency initiative coordinated across the FDA, NCI, NCAT...
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In the space of five days, the FDA reversed its position on two rare disease gene therapies it had previously rejected. On June 17, the agency agreed that UniQu...
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Most genetic medicine is built for populations large enough to support a clinical trial. N-of-1 medicine is built for the opposite case: a single patient whose ...
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On June 2, 2026, the FDA released draft guidance that could reshape how gene therapies reach patients. The document, "Leveraging Prior Knowledge in the Developm...
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Bipolar disorder has long been recognized as highly heritable, yet the biology underlying the condition has remained difficult to resolve. The genetics are comp...
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Finding patients who qualify for a clinical trial has always been one of the hardest parts of running one. In liver disease, particularly MASLD, it is especiall...
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