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    pharmacological chaperones
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    Journal Articles

    New Derivatives of L-Idonojirimycin Working As Pharmacological Chaperones for the Treatment of Gaucher Disease

    Pilar Alfonso, PhD, Javier Gervas, Vanesa Andreu, PhD, Joaquin Navascues, PhD, Francisca Sanchez-Jimenez, Prof, Miguel Pocovi, Prof, Carmen Ortiz-Mellet, Prof, Jose M Garcia-Fernandez, Prof, Pilar Giraldo, MD, PhD
    Journal: Blood
    Blood (2012) 120 (21): 3270.
    https://doi.org/10.1182/blood.V120.21.3270.3270
    Published: 2012
    Journal Articles

    Pharmacological Chaperone Therapy for the Treatment of Gaucher Disease: Results of Phase 1 Clinical Trials and a Clinical Ex Vivo Response Study with a Survey of Blood Markers for 53 Gaucher Patients.

    Neal J. Weinreb, MD, G.A. Grabowski, MD, G.M. Pastores, MD, P.M. Fernhoff, MD, P.B. Kaplan, MD, D. Palling, PhD, C.W. Pine, B.E. Raines, F. Insinga, D.J. Lockhart, PhD, H.V. Do, PhD, B.A. Wustman, PhD
    Journal: Blood
    Blood (2007) 110 (11): 2404.
    https://doi.org/10.1182/blood.V110.11.2404.2404
    Published: 2007

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