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Funding Awarded

2 awards

  • Ex vivo autologous stem cell gene therapy for Multiple Sulfatase Deficiency

    Multiple Sulfatase Deficiency (MSD) is an inherited lysosomal storage disorder (LSD) that predominately involves the brain, bones, and skin. The disease is due to mutations in SUMF1, a gene that provides the instructions for making an important enzyme called formylglycine generating enzyme (FGE). The FGE enzyme activates all the...

  • Evidence Synthesis Ireland

    Health care decisions should be based on the combination of the full amount of information that is available rather than relying on the convenient selection of one or more individual pieces of information, or studies. Evidence synthesis methods seek to establish the overall balance of information on a given topic, and are based on...