Archives: 2021-01-08

Orchard Therapeutics with research alliance partners Fondazione Telethon and Ospedale San Raffaele Milan Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)

First gene therapy to receive full EU marketing authorization for eligible MLD patients. One-time treatment with Libmeldy has been shown to preserve motor and cognitive function. Orchard Therapeutics, a global gene therapy leader, and its research alliance partners Fondazione Telethon and Ospedale San Raffaele, today announced that the European Commission (EC) granted full (standard) market authorization

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DNA-editing method shows promise to treat mouse model of progeria

Using a recently developed DNA base-editing technique, researchers correct accelerating aging disorder. Researchers have successfully used a DNA-editing technique to extend the lifespan of mice with the genetic variation associated with progeria,  a rare genetic disease that causes extreme premature aging in children and can significantly shorten their life expectancy. The study was published in the journal Nature, and was

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Stem cell treatment corrects skull shape and restores brain function in mouse model of childhood disorder

Scientists regenerate parts of the skull affected by craniosynostosis, a common birth defect. Using stem cells to regenerate parts of the skull, scientists corrected skull shape and reversed learning and memory deficits in young mice with craniosynostosis, a condition estimated to affect 1 in every 2,500 infants born in the United States, according to the

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Early IgG titer greater than 1:1000 against SARS-CoV-2 spike protein Plasma Therapy to Prevent Severe Covid-19 in Older Adults

Therapies to interrupt the progression of early coronavirus disease 2019 (Covid-19) remain elusive. Among them, convalescent plasma administered to hospitalized patients has been unsuccessful, perhaps because antibodies should be administered earlier in the course of illness. In this paper, published on NEJM, the Authors conducted a randomized, double-blind, placebo-controlled trial of convalescent plasma with high

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The neutrophil-to-lymphocyte ratio determines clinical efficacy of corticosteroid therapy in patients with COVID-19

Corticosteroid therapy is now recommended as a treatment in patients with severe COVID-19. But one key question is how to objectively identify severely ill patients who may benefit from such therapy. In a paper just published on Cell Metabolism, 12,862 COVID-19 cases from 21 hospitals in Hubei Province were equally assigned to a training and

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Mutations arising in SARS-CoV-2 spike on sustained human-to-human transmission and human-to-animal passage

The proximal origins of SARS-CoV and MERS-CoV from civets and camels, respectively, are well documented. Few genetic changes in these viruses are required for the interspecies transfers to humans. While precise details and timing of the evolutionary pathways remain to be elucidated, it is also apparent that SARS-CoV-2 emerged from the Sarbecovirus subgenus of the

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SARS-CoV-2 Variants

SARS-CoV-2, the virus that causes COVID-19, has had a major impact on human health globally; infecting a large number of people; causing severe disease and associated long-term health sequelae; resulting in death and excess mortality, especially among older and vulnerable populations; interrupting routine healthcare services; disruptions to travel, trade, education and many other societal functions;

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New SARS-CoV-2 variants described by researchers of University College of London and Cambridge University

Does it spread more easily? Make people sicker? Mean that treatments and vaccines won’t work? Questions are multiplying as fast as new variants of the coronavirus, especially the one moving through England and now popping up in the U.S. and other countries. Scientists say there is reason for concern and more to learn but that

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