When a couple loses a pregnancy, and especially when the same couple loses multiple pregnancies, doctors are often at a loss to explain why. For about 40 to 60 percent of couples with recurrent miscarriage (RM), the condition remains unexplained, even after costly testing. Chromosomal abnormalities — rearrangements of large chunks of DNA — in the genomes of one or…
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Disc Medicine, headquartered in Cambridge, Massachusetts, announced the completion of a $50 million Series A financing. The round was led by Novo Holdings along with Access Biotechnology and founding investor Atlas Venture. The company also announced today that it had inked an exclusive deal with AbbVie for a series of hemojuvelin antagonist monoclonal antibodies. Disc Medicine is focused on hepcidin biology to restore red blood cell…
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It’s not every day that scientists get to publish a paper on Halloween about a skeleton, but that’s what the laboratories of Alan Brown in the Blavatnik Institute at Harvard Medical School and Rui Zhang at Washington University in St. Louis just did. As reported in the Oct. 31 print issue of Cell, the collaborating teams mapped out the atomic structures of significant components of…
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Promising Results Reported in Tay-Sachs Gene Therapy Trial

Researchers have obtained the first signs of clinical benefit in an early-stage gene therapy trial for Tay-Sachs disease, according to a presentation at the European Society of Gene & Cell Therapy (ESGCT) annual conference last week. Terence R. Flotte, MD, executive deputy chancellor, provost, and dean of the University of Massachusetts (UMass) School of Medicine, presented the results in Barcelona.…
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CAMBRIDGE, Mass. and DUBLIN, Oct. 30, 2019 /PRNewswire/ — Biogen Inc. (Nasdaq: BIIB) and Alkermes plc (Nasdaq: ALKS) today announced that the U.S. Food and Drug Administration (FDA) approved VUMERITY™ (diroximel fumarate), a novel oral fumarate with a distinct chemical structure, for the treatment of relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. Biogen holds the exclusive, worldwide license…
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The investigational KRAS G12C inhibitor MRTX849 yielded clinical responses in patients with non-small cell lung cancer (NSCLC) and colorectal cancer harboring KRAS G12C mutations, according to data from a phase I clinical trial presented today at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics by Dana-Farber Cancer Institute researchers. Data demonstrating the efficacy of MRTX849 in preclinical studies and in two patients…
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Oxford BioDynamics has announced that the first patient has been enrolled in the REFINE-ALS clinical study, designed to identify and measure specific biomarkers and clinical assessments in up to 300 people with amyotrophic lateral sclerosis (ALS) who are taking Radicava (edaravone). The study includes assessments of biomarkers for oxidative stress, inflammation, neuronal and muscle injury. Biomarkers will be measured prior…
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MIT chemists have devised a way to synthesize polymers that can break down more readily in the body and in the environment. A chemical reaction called ring-opening metathesis polymerization, or ROMP, is handy for building novel polymers for various uses such as nanofabrication, high-performance resins, and delivering drugs or imaging agents. However, one downside to this synthesis method is that…
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Autism spectrum disorders affect one in 59 American children by age eight. With no known quantitative biological features, autism diagnoses are currently based on expert assessments of behavioral symptoms, including impaired social skills and communication, repetitive behaviors and restricted interests. In a paper published in Annals of Neurology, Matthew P. Anderson, MD, PhD, a physician-scientist at Beth Israel Deaconess Medical Center…
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