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Onasemnogene abeparvovec-xioi (Zolgensma): Approval strategy with manipulated data?

August 13, 2019 – Onasemnogene abeparvovec-xioi (Zolgensma) is an adeno-associated virus vector-based gene therapy indicated for the treatment of pediatric patients less than 2 years of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.

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Gene therapy: Zolgensma to treat spinal muscular atrophy

May 29, 2019 – Spinal muscular atrophy (SMA) is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting. The severity of symptoms and age of onset varies by the type. Some types are apparent at or before birth while others are not apparent until adulthood. 

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Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy

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  • New study shows how cancer survivors develop opioid addictions November 22, 2019
    Opioids play an important role in how cancer patients manage pain, but the ongoing opioid epidemic has raised concerns about their potential for abuse. A new study in the Journal of the National Cancer Institute reveals that several factors are associated with a risk for persistent opioid use, including younger age, white race, unemployment at […]
  • Lack of sleep may explain why poor people get more heart disease November 22, 2019
    Insufficient sleep is one reason why disadvantaged groups have more heart disease. That's the finding of a study published today in Cardiovascular Research, a journal of the European Society of Cardiology (ESC).
  • Women raised in poor neighborhoods face an increased risk of intimate partner violence November 21, 2019
    Women who spend longer periods of their early lives in less affluent neighbourhoods are at greater risk of experiencing violence during their early adulthoods at the hands of their intimate partners, finds a new study published in Epidemiology.
  • A review of single molecule-based electronic devices November 21, 2019
    In a paper to be published in the forthcoming issue in NANO, a group of researchers from the Shenyang Jianzhu University in China provide an overview of single molecule electronic devices, including molecular electronic devices and electrode types. Future challenges are described in the hopes of attracting more experts from different fields to participate in […]
  • Wolfe Creek Crater younger than previously thought November 21, 2019
    Wolfe Creek Crater, one of the world's largest meteorite craters, is much younger than previously thought.
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