The investigational drug evinacumab reduced triglycerides in patients with severe hypertriglyceridemia (sHTG) and a history of hospitalizations for acute pancreatitis in a phase 2 global study led by Mount Sinai. The fully ...
May 16, 2021
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University of Dundee researchers have discovered a new neurodevelopmental disease caused by genetic mutations and helped explain why patients with these mutations suffer this condition.
Apr 23, 2021
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Apic Bio, a gene therapy company developing treatment options for patients with rare genetic diseases and co-founded by UMass Medical School's Robert H. Brown Jr., DPhil, MD, announced that the FDA has cleared its investigational ...
Apr 22, 2021
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A gene therapy protects eye cells in mice with a rare disorder that causes vision loss, especially when used in combination with other gene therapies, shows a study published today in eLife.
Apr 13, 2021
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New research led by a professor at NUI Galway is set to change how doctors treat some patients with high blood pressure—a condition that affects more than one in four men and one in five women.
Apr 13, 2021
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In 2014, two years after her Nobel Prize-winning invention of CRISPR-Cas9 genome editing, Jennifer Doudna thought the technology was mature enough to tackle a cure for a devastating hereditary disorder, sickle cell disease, ...
Mar 31, 2021
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The COVID vaccine rollout is underway in Australia, with people in phase 1b now eligible to be vaccinated.
Mar 29, 2021
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For the first time researchers from Hebrew University of Jerusalem, Radboudumc, Maastricht UMC+ and international colleagues have gained insight into the "hidden genetic defects" of the general European population. This is ...
Mar 19, 2021
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Researchers from the Mitochondrial Medicine Frontier Program at Children's Hospital of Philadelphia (CHOP) have demonstrated how one combination of therapies may be beneficial for patients with mitochondrial respiratory chain ...
Mar 2, 2021
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In African Americans, the genetic risk landscape for inflammatory bowel disease (IBD) is very different from that of people with European ancestry, according to results of the first whole-genome study of IBD in African Americans. ...
Feb 19, 2021
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