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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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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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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Spinal Muscular Atrophy: Single-Dose Gene-Replacement Therapy

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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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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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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 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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