US FDA grants Rare Pediatric Disease Designation to gene therapy OTOF-GT for the treatment of otoferlin gene-mediated hearing loss

OTOF-GT targets the restoration of hearing in people living with otoferlin deficiency Sensorion, a pioneering clinical-stage biotechnology company which specializes in the development of novel therapies to restore, treat and prevent hearing loss disorders, announces that the US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to the Company’s lead therapy gene

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Which COVID Vaccine You Get Can Impact Myocarditis Risk: study published in the Journal of the American College of Cardiology

Study shows higher rates of heart inflammation with Moderna vs. Pfizer, but overall risk still very low Incidence of myocarditis, pericarditis or myopericarditis is two- to threefold higher after a second dose of the Moderna Spikevax COVID-19 vaccine when compared to the Pfizer BioNTech COVID-19 vaccine; however, overall cases of heart inflammation with either vaccine

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COVID-19 deaths in children and young people in England, March 2020 to December 2021: an active prospective national surveillance study

A new study conducted in England shows that the risk of death due to COVID-19 remains very low for children and young people, and most deaths occur in those with underlying health conditions. A new study conducted in England shows that the risk of death due to COVID-19 remains very low for children and young

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New drug can successfully treat patients typically resistant to high blood pressure treatment

A new drug called Baxdrostat has been shown to significantly reduce high blood pressure (hypertension) in patients who may not respond to current treatments for the condition, according to results from a phase II trial led jointly by a Queen Mary University of London researcher and colleagues at CinCor Pharma, USA. Published in the New England

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Intravenous iron improved long-term outcomes for people with heart failure and iron deficiency

Research Highlights: Repeated intravenous (IV) administration of iron reduced the risk of hospitalization for heart failure and cardiovascular death in people with heart failure and iron deficiency, according to a clinical trial in the United Kingdom. Treatment with IV iron was safe and well-tolerated, and people who received it reported improved well-being based on quality-of-life

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Novel CRISPR/Cas9-based gene-editing therapy shows promise for patients with transthyretin amyloid cardiomyopathy

A single IV infusion of NTLA-2001, a novel CRISPR/Cas9-based gene editing therapy, significantly reduced circulating transthyretin (TTR) protein levels in patients with ATTR amyloid cardiomyopathy, a progressive and fatal cause of heart failure, according to late-breaking research presented today at the American Heart Association’s Scientific Sessions 2022. The meeting, held in person in Chicago and

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Organoids Reveal How SARS-CoV-2 Damages Brain Cells — and a Potential Treatment

COVID-19 infections can result in long-lasting neurological symptoms; new research suggests an already approved anti-viral may inhibit viral replication and rescue impaiOrganoids Reveal How SARS-CoV-2 Damages Brain Cells — and a Potential Treatmentred neurons Using human brain organoids, an international team of researchers, led by scientists at University of California San Diego School of Medicine

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Over 350 Multidisciplinary Experts from More than 100 Countries Reach Consensus on How to End COVID-19 as a Public Health Threat

A new global COVID-19 study provides actionable recommendations to end the public health threat without exacerbating socio-economic burdens or putting the most vulnerable at greater risk SARS-CoV-2 continues to circulate among us. Although some governments have moved on, a new study published today in the journal Nature says that specific efforts and resources are still required to save

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Study shows GABA, an inexpensive and readily available chemical, reduces disease severity, lung viral load and death in SARS-CoV-2-infected mice

Preclinical studies in mice that model human COVID-19 suggest that an inexpensive, readily available amino acid might limit the effects of the disease and provide a new off-the-shelf therapeutic option for infections with SARS-CoV-2 variants and perhaps future novel coronaviruses. A team led by researchers at the David Geffen School of Medicine at UCLA report that an

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