Researchers at the University of Toronto's Donnelly Center for Cellular and Biomolecular Research have found nearly one million new exons—stretches of DNA that are expressed in mature RNA—in the human genome.
Feb 9, 2024
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Researchers at the National Institutes of Health detected abnormal proteins in the spinal fluid of people with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), which could help improve diagnosis of these ...
Jan 31, 2024
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A group of Ludwig Maximilian University scientists led by Eckhard Wolf has developed a porcine DMD model with a mutation that mimics the hallmarks of the human disease but develops them in an accelerated mode. The study is ...
Jul 13, 2023
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A Phase II clinical trial of poziotinib for non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 mutations, led by researchers at The University of Texas MD Anderson Cancer Center, found ...
Jul 11, 2022
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Up to 45% of patients with the most common inherited neuromuscular disease could benefit from a new "cocktail" drug being developed at the University of Alberta, according to research published this week in the Proceedings ...
Feb 23, 2022
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This first-in-human study of golodirsen showed its long-term safety and biologic activity in patients with Duchenne Muscular Dystrophy (DMD). The approved exon-skipping therapy is designed to enable the production of functional ...
Nov 30, 2021
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Tagrisso (osimertinib) received approval as an adjuvant treatment for patients with non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R mutations, the U.S. Food and Drug ...
Dec 23, 2020
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One of the most recognizable characteristics of autism is an amazing diversity of associated behavioral symptoms. Clinicians view autism as a broad spectrum of related disorders, and the origin of the disease's heterogeneity ...
Dec 22, 2020
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A targeted therapy called capmatinib can provide significant benefits to patients who have advanced lung cancer with specific gene mutations, according to recently published results from a phase two clinical trial. The trial, ...
Sep 8, 2020
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A small-molecule drug is the first to preserve hearing in a mouse model of an inherited form of progressive human deafness, report investigators at the University of Iowa, Iowa City, and the National Institutes of Health's ...
Jun 28, 2018
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