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    Journal Articles

    Breakthroughs in Preclinical Development of Ataluren (PTC124) As Therapeutic Option for Patients Affected By Shwachman-Diamond Syndrome: Towards the First Clinical Trial

    Valentino Bezzerri, PhD, Antonio Vella, PhD, Elisabetta D'Aversa, PhD, Martina Api, PhD, Marisole Allegri, MSc, Elena Marinelli Busilacchi, PhD, Giovanna D'Amico, BSc, PhD MSc, Roberto Gambari, PhD, Claudio Sorio, MD PhD, Benedetta Fabrizzi, MD, Vincenzo Bronte, MD, Antonella Poloni, PhD MD, Monica Borgatti, PhD, Marco Cipolli, MD
    Journal: Blood
    Blood (2019) 134 (Supplement_1): 451.
    https://doi.org/10.1182/blood-2019-127866
    Published: 2019
    Journal Articles

    Nonsense Suppression Approaches in Treating Hemophilia

    Yi-Lin Liu, Paris Margaritis, Fayaz Khazi, Harre Downey, Stephan Kadauke, Nicole Hasbrouck, Josephine Sheedy, Ellen Welch, Marla Weetall, Katherine A. High, MD
    Journal: Blood
    Blood (2008) 112 (11): 512.
    https://doi.org/10.1182/blood.V112.11.512.512
    Published: 2008

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