New gene therapy breakthrough may help in the treatment of children with hearing loss

A new study presents an innovative treatment for deafness, based on the delivery of genetic material into the cells of the inner ear. The genetic material ‘replaces’ the genetic defect and enables the cells to continue functioning normally. They maintain that this novel therapy could lead to a breakthrough in treating children born with various mutations that eventually cause deafness.

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RedHill’s P2/3 COVID-19 candidate Opaganib reduces ARDS-related blood clotting

RedHill Biopharma Ltd, a specialty biopharmaceutical company, announced promising preliminary results from a preclinical study with opaganib, a novel, orally administered sphingosine kinase 2 (SK2) selective inhibitor, administered at 250mg/kg, demonstrating a reduction of thrombosis (blood clotting) in an acute respiratory distress syndrome (ARDS) – a preclinical animal model designed to measure thrombotic risks.

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MRI during treatment could improve paediatric proton therapy

Identifying anatomic changes shown on MRI scans during a course of pencil-beam proton therapy, and adapting treatment plans accordingly, could improve the treatment quality, effectiveness and safety. In a retrospective study, researchers at St. Jude Children’s Research Hospital determined that 27% of paediatric patients, the majority with brain tumours, would have benefited from mid-treatment plan changes.

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Pfizer vaccine gets green light from FDA committee

An independent committee of experts recommended Thursday that the Food and Drug Administration authorize the Pfizer and BioNTech COVID-19 vaccine for people over the age of 16. Now, the FDA will decide whether to follow that recommendation. The agency could announce a decision within days. If they do authorize the vaccine, health care workers could begin receiving the shots almost immediately.

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