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Wed, Jul 16, 2025

Rare diseases

Home » Topics » Science » Rare diseases
  • Research mouse polygonal wireframe
    May 23, 2025
    By Mar de Miguel

    From mice to big animal models in gene therapy for rare diseases

    The lack of animal models that mimic human disease impedes the study of many pathologies that still lack treatment beyond symptom relief. This is what has happened so far with PURA syndrome, a rare disorder affecting brain development for which a mouse model has finally been developed. Other times, small and large models exist, but an effective treatment remains elusive, as is the case with Krabbe disease, a fatal disease in children that could be prevented with the advances in gene therapy.
  • Cobra protein hero
    Jan. 22, 2025
    By Mar de Miguel

    An antidote to cobra venom designed with AI

    Following Nobel Prize-winning chemist David Baker’s recipe for cooking an antidote to cobra venom using artificial intelligence (AI) could be faster and more effective than currently available methods. The ingredients and steps can be found in a new study published by the University of Washington scientist in collaboration with the Technical University of Denmark. They are ready for the next steps in preclinical trials.
  • Cobra protein hero
    Jan. 21, 2025
    By Mar de Miguel

    An antidote to cobra venom designed with AI

    Following Nobel Prize-winning chemist David Baker’s recipe for cooking an antidote to cobra venom using artificial intelligence (AI) could be faster and more effective than currently available methods. The ingredients and steps can be found in a new study published by the University of Washington (UW) scientist in collaboration with the Technical University of Denmark. They are ready for the next steps in preclinical trials.
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    By Nuala Moran

    The EMA’s Shambolic Handling of Glybera

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