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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 type of taste cell discovered in taste buds August 13, 2020
    Our mouths may be home to a newly discovered set of multi-tasking taste cells that—unlike most known taste cells, which detect individual tastes—are capable of detecting sour, sweet, bitter and umami stimuli. A research team led by Kathryn Medler at the University at Buffalo reports this discovery in a study published 13th August in PLOS […]
  • Researchers discover genetic link to predict positive response to immunotherapy in patient August 13, 2020
    A Singapore team led by clinician-scientists and researchers from the National Cancer Centre Singapore (NCCS) discovered a genetic link to better predict treatment response for relapsed/refractory patients with natural-killer T-cell lymphoma (NKTCL), a highly aggressive form of blood cancer. The team performed whole-genome sequencing, to identify mutation in PD-L1 gene as a reliable biomarker to […]
  • Evolution and everyday stress have led to disproportionate suffering among women August 13, 2020
    We're 100% behind knowledge-based research, but sometimes you really do have to question evolution!
  • A cancer mystery more than 40 years old is solved thanks to epigenetics August 12, 2020
    Before the first oncogene mutations were discovered in human cancer in the early 1980s, the 1970s provided the first data suggesting alterations in the genetic material of tumors. In this context, the prestigious journal Nature published in 1975 the existence of a specific alteration in the transformed cell: an RNA responsible for carrying an amino […]
  • Combining genetic information with EMRs to pinpoint childhood epilepsies August 11, 2020
    A team of researchers at Children's Hospital of Philadelphia (CHOP) affiliated with the CHOP Epilepsy Neurogenetics Initiative (ENGIN) further bridged the gap between genomic information and clinical outcome data by systematically linking genetic information with electronic medical records, focusing on how genetic neurological disorders in children develop over time. The findings were published today in […]
  • Liquid crystals that can replace color shifting ink in preventing counterfeiting August 14, 2020
    A research team in Korea has developed a material that may potentially replace color shifting ink in prevention of forgery of bank notes, ID cards, and so on. A team headed by Dr. Sang-seok Lee from the Functional Composite Material Research Center of the Korea Institute of Science and Technology successfully developed a technology to […]
  • Sounds of action: Using ears, not just eyes, improves robot perception August 14, 2020
    People rarely use just one sense to understand the world, but robots usually only rely on vision and, increasingly, touch. Carnegie Mellon University researchers find that robot perception could improve markedly by adding another sense: hearing.
  • Research captures how human sperm swim in 3D August 13, 2020
    Using state-of-the-art 3D microscopy and mathematics, Dr Hermes Gadêlha from the University of Bristol, Dr Gabriel Corkidi and Dr Alberto Darszon from the Universidad Nacional Autonoma de Mexico, have reconstructed the movement of the sperm tail in 3D with high-precision.
  • Global deaths due to smokeless tobacco are up by a third, according to new study August 13, 2020
    The number of deaths globally due to smokeless tobacco has gone up by a third in 7 years to an estimated 350,000 people, a new study suggests.
  • Employers reject transgender people August 13, 2020
    Employers in Sweden more often reject job applications from transgender people -- especially in male-dominated occupations. Moreover, transgender people face discrimination from two different grounds for discrimination. This is according to a study from Linköping University that was recently published in the journal Labour Economics.
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