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ANSM et le thérapie génique: accès précoce aux premiers médicaments innovants “CAR T-Cells”

27 juillet 2018 –  Cette point d’information vient de l’Agence nationale de sécurité du médicament et des produits de santé (ANSM) concernant l’accès précoce aux premiers médicaments innovants “CAR T-Cells” dans le traitement de certains cancers hématologiques.

En juin 2018, l’Agence européenne des médicaments (EMA) a rendu un avis positif pour l’autorisation de mise sur le marché des deux premiers médicaments de thérapie génique Kymriah et Yescarta appelés “CAR T-Cells”,

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  • Distinct social profile and high ASD risk, 3q29 deletion survey finds juillet 19, 2019
    A survey of 93 people with 3q29 deletion syndrome reveals a distinct pattern of social disability and anxiety, even without a diagnosis of autism spectrum disorder. The results were published online in Molecular Autism on July 16.
  • Study sheds light on the darker parts of our genetic heritage juillet 19, 2019
    More than half of our genome consists of transposons,DNA sequences that are reminiscent of ancient, extinct viruses.Transposons are normally silenced by a process known as DNA methylation, but their activation can lead to serious diseases. Very little is known about transposons but researchers in an international collaboration project have now succeeded for the first time […]
  • Molecular cause for severe multi-organ syndrome juillet 19, 2019
    Three unrelated families on three continents (from continental Portugal, the United States and Brazil), all with healthy ancestors, had children with a very rare multi-organ condition that causes early-onset retinal degeneration, sensorineural hearing loss, microcephaly, intellectual disability, and skeletal dysplasia with scoliosis and short stature.
  • Researchers determine epigenetic origin of docetaxel-resistant breast cancer juillet 19, 2019
    Researchers at the Bellvitge Biomedical Research Institute (IDIBELL) have published a study in Molecular Cancer Research in which they identify methylation patterns associated with different subtypes of breast cancer, and a subclassification of the group of "triple negatives," a breast cancer type typically associated with poor prognosis. In addition, they identified changes in DNA methylation […]
  • Researchers report the function of reverse-twisting DNA juillet 19, 2019
    Normally, the two strands of the DNA double helix wind around each other in a right-handed spiral. However, there is another conformation called Z-DNA in which the strands twist to the left. The function of Z-DNA has remained a mystery since its discovery. A newly published paper unambiguously establishes that the Z-conformation is key to […]
  • Study sheds light on the darker parts of our genetic heritage juillet 19, 2019
    More than half of our genome consists of transposons, DNA sequences that are reminiscent of ancient, extinct viruses. Transposons are normally silenced by a process known as DNA methylation, but their activation can lead to serious diseases. Very little is known about transposons but researchers in an international collaboration project have now succeeded for the first time […]
  • Flexible user interface distribution for ubiquitous multi-device interaction juillet 19, 2019
    KAIST researchers have developed mobile software platform technology that allows a mobile application (app) to be executed simultaneously and more dynamically on multiple smart devices. Its high flexibility and broad applicability can help accelerate a shift from the current single-device paradigm to a multiple one, which enables users to utilize mobile apps in ways previously […]
  • Atomically precise models improve understanding of fuel cells juillet 19, 2019
    Simulations from researchers in Japan provide new insights into the reactions occurring in solid-oxide fuel cells by using realistic atomic-scale models of the electrode active site based on microscope observations instead of the simplified and idealized atomic structures employed in previous studies. This better understanding of how the structures in the cells affect the reactions […]
  • Predicting long-term risk of death from chest X-rays juillet 19, 2019
    Researchers in this study looked at whether a computing system that analyzed data from thousands of chest X-rays of smokers and nonsmokers and developed a risk score could predict long-term risk of death.
  • Ivacaftor may reduce common infections in patients with CF juillet 19, 2019
    Patients with cystic fibrosis who take ivacaftor appear to have fewer respiratory infections over time than those not taking the drug, according to new research published online in the Annals of the American Thoracic Society.
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