Blog Archives

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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Voretigene Neparvovec-Rzyl (Luxturna): Gene therapy to treat inherited vision loss

December 20, 2017 –  The American Food & Drug Administration (FDA) has just approved  a novel gene therapy to treat patients with a rare form of inherited vision loss. Voretigene Neparvovec-Rzyl (Luxturna) is the first directly administered gene therapy approved to target a disease caused by mutations in a specific human gene (i.e.,

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Marsili Syndrome: A mutation in the ZFHX2 gene leaves Italian family painless

December 21, 2017 – No pain on gain. This headline might not at all apply for this Italian family. In a paper just published in the Journal Brain,

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Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy

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The first gene therapy FDA approved in the US

September 3, 2017 – For a very first time, the American Food & Drug Administration (FDA) has approved a gene therapy for the United States (US) in that it approved CAR T-cell therapy to treat certain children and young adults with B-cell acute lymphoblastic leukemia (B-cell ALL).

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Axicabtagene Ciloleucel (Yescarta) gene therapy for diffuse large B-cell lymphoma

October 19, 2017 – The American  Food & Drug Administration (FDA) just approved Axicabtagene Ciloleucel (Yescarta), a cell-based gene therapy, to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment.

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New hope for children with the ultra-rare immune disorder ADA-SCID: Gene therapy Strimvelis recommended for approval by EMA’s CHMP

April 02, 2016 – The European Medicines Agency (EMA) has recommended granting a marketing authorisation in the European Union (EU) for a new gene therapy for the treatment of patients with adenosine-deaminase-deficient severe combined immunodeficiency (ADA-SCID), who have no matching donor for a stem cell transplant.

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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 […]
  • 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.
  • How a protein stops cells from attacking their own DNA August 13, 2020
    Scientists at EPFL have demonstrated the mechanism that allows cells to fight off viral DNA without triggering an immune response against their own genetic material.
  • 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.
  • A watershed moment for US water quality August 13, 2020
    A new federal rule that determines how the Clean Water Act is implemented leaves millions of miles of streams and acres of wetlands unprotected based on selective interpretation of case law and a distortion of scientific evidence, researchers say in a new publication.
  • Study: Medical marijuana associated with fewer hospitalizations for individuals with SCD August 13, 2020
    Individuals with sickle cell disease (SCD) who receive medical marijuana to treat pain may require fewer visits to the hospital, according to a new study in Blood Advances. Adults with SCD who requested and obtained medical marijuana were admitted to the hospital less frequently than those who did not obtain it.
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