(HealthDay)—Use of genomic sequencing yields an increase in genomic diagnoses across a spectrum of rare diseases, according to a study published in the Nov. 11 issue of the New England Journal of Medicine.
Dec 3, 2021
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A team, led from Great Ormond Street Hospital (GOSH), UCL Great Ormond Street Institute of Child Health (UCL GOS ICH) and the Istituto Zooprofilattico Sperimentale delle Venezie (Legnaro, Italy), has built on recent advances ...
Dec 2, 2021
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Autoimmune conditions like Type 1 diabetes, multiple sclerosis and rheumatoid arthritis arise when an immune system mistakenly attacks its own body's proteins, cells and organs. Not only do these conditions make the body ...
Nov 29, 2021
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Two-year-old Haoyang has likely just months to live—but the only medicine that can help his rare genetic condition is not found anywhere in China and closed borders due to the COVID-19 pandemic mean he cannot travel for ...
Nov 23, 2021
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Riluzole, a drug approved to treat amyotrophic lateral sclerosis (ALS), a disease affecting nerve cells controlling movement, could slow the gradual loss of a particular brain cell that occurs in Niemann-Pick disease type ...
Nov 19, 2021
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Denmark's Novo Nordisk, the world's leading insulin maker, said Thursday it will buy US-based Dicerna Pharmaceuticals, a developer of RNA interference technology which targets genes that cause disease.
Nov 18, 2021
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Precision medicine requires big data. In order to improve the treatment of individuals with cancer, or to understand rare diseases, scientists and clinicians, as well as AI technologies require access to larger sets of health ...
Nov 18, 2021
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Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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Valsartan, an angiotensin II receptor blocker drug, delayed disease progression and improved cardiac structure and function in patients with early-stage hypertrophic cardiomyopathy, according to results from a recent clinical ...
Nov 17, 2021
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Pancreatic cancer forms in the cells of the pancreas, an organ in the abdomen that releases enzymes that aid digestion and produces hormones that help manage our blood sugar levels. Pancreatic cancer is a rare disease with ...
Nov 11, 2021
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