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Hunter syndrome

This page shows the latest Hunter syndrome news and features for those working in and with pharma, biotech and healthcare.

Trial dents confidence in Sangamo’s gene-editing tech

Trial dents confidence in Sangamo’s gene-editing tech

Hunter syndrome was able to induce expression of the IDS enzyme that is deficient in the disease. ... was able to induce expression of the IDUA enzyme missing in the disease, which is also known as Hurler syndrome.

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  • 2019: CRISPR and therapeutic gene editing comes of age 2019: CRISPR and therapeutic gene editing comes of age

    The phase 1/2 CHAMPIONS study did show that a single injection of SB-913 for the rare genetic disease mucopolysaccharidosis type II (MPS II) or Hunter syndrome was able to

  • Deal Watch table for July 2014 Deal Watch table for July 2014

    360. Sigma-Tau/ Jazz Pharmaceuticals. Regaining rights. Defibrotide for severe hepatic veno-occlusive disease (approved EU/ NDA US)    . 250. ArmaGen/ Shire   . Licence, collaboration   . AGT-182, enzyme replacement therapy for Hunter

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  • TVF Communications shortlisted for two awards at PMEA 2016!

    For both target diseases, Hunter Syndrome(MPS II) and Hidradenitis Suppurativa (HS), a timely diagnosis is imperative, as the early symptoms are often mistaken for far more common and far less ... refer to a geneticist if the syndrome is suspected.

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