Researchers at the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health (NIH), have developed the Families Sharing Health Assessment and
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For children with spinal muscular atrophy (SMA), gene therapy with onasemnogene abeparvovec (Zolgensma, Novartis) provides long-lasting benefits with a favorable safety profile, new long-term follow-up
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Editor’s note: Find the latest COVID-19 news and guidance in Medscape’s Coronavirus Resource Center. In a typical year, the surveillance team at the University of
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Scientists at UC San Francisco, UC Berkeley and UCLA have received U.S. Food and Drug Administration approval to jointly launch an early phase, first-in-human clinical
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An investigational gene therapy, elivaldogene autotemcel (eli-cel, Bluebird Bio), appears to hold at bay the devastating neurodegenerative disease cerebral adrenoleukodystrophy (ALD). Two years after
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As the fight against COVID-19 continues, scientists have turned to an unlikely source for a potentially effective treatment: tiny antibodies naturally generated by llamas. While
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CER-001 (Abionyx Pharma), an apolipoprotein A1 (apoA-1)–containing high-density lipoprotein (HDL) mimetic, may help restore kidney function and vision for patients suffering from a rare genetic
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Editor’s note: Find the latest COVID-19 news and guidance in Medscape’s Coronavirus Resource Center. The US Food and Drug Administration (FDA) on Monday updated its
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Skip to Energy transformation in metabolism Key biomolecules Thermodynamics of metabolism Metabolism control Metabolism disorders and manipulation Evolution History Energy transformations in metabolism The energy
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The isolation of DNA was successfully carried out in 1869. However, its sequencing had to wait until the Human Genome Project was completed in 2003,
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