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There are 20710 results for: content related to: Treating lysosomal storage diseases with pharmacological chaperones: from concept to clinics

  1. New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders

    Wiley Interdisciplinary Reviews: Nanomedicine and Nanobiotechnology

    Volume 2, Issue 2, March/April 2010, Pages: 189–204, Silvia Muro

    Version of Record online : 28 JAN 2010, DOI: 10.1002/wnan.73

  2. You have full text access to this OnlineOpen article
    Disease models for the development of therapies for lysosomal storage diseases

    Annals of the New York Academy of Sciences

    Volume 1371, Issue 1, May 2016, Pages: 15–29, Miao Xu, Omid Motabar, Marc Ferrer, Juan J. Marugan, Wei Zheng and Elizabeth A. Ottinger

    Version of Record online : 4 MAY 2016, DOI: 10.1111/nyas.13052

  3. Lysosomal Storage Disorders: Gene Therapy

    Standard Article


    Mario A Cabrera-Salazar and John A Barranger

    Published Online : 27 JAN 2006, DOI: 10.1038/npg.els.0005754

  4. Poster abstracts

    Acta Paediatrica

    Volume 94, Issue s447, March 2005, Pages: 95–133,

    Version of Record online : 2 JAN 2007, DOI: 10.1111/j.1651-2227.2005.tb02120.x

  5. You have full text access to this OnlineOpen article
    New therapeutic approaches for Krabbe disease: The potential of pharmacological chaperones

    Journal of Neuroscience Research

    Volume 94, Issue 11, November 2016, Pages: 1203–1219, Samantha J. Spratley and Janet E. Deane

    Version of Record online : 17 SEP 2016, DOI: 10.1002/jnr.23762

  6. You have full text access to this OnlineOpen article
    Transcription factor EB (TFEB) is a new therapeutic target for Pompe disease

    EMBO Molecular Medicine

    Volume 5, Issue 5, May 2013, Pages: 691–706, Carmine Spampanato, Erin Feeney, Lishu Li, Monica Cardone, Jeong-A Lim, Fabio Annunziata, Hossein Zare, Roman Polishchuk, Rosa Puertollano, Giancarlo Parenti, Andrea Ballabio and Nina Raben

    Version of Record online : 18 APR 2013, DOI: 10.1002/emmm.201202176

  7. You have full text access to this OnlineOpen article
    Can stapedius reflex testing objectively measure muscle function in Pompe patients?

    Clinical Case Reports

    Volume 3, Issue 11, November 2015, Pages: 937–941, Max J. Hilz, Ulrich Hoppe, Sebastian Moeller, Ruihao Wang and Julia Koehn

    Version of Record online : 28 SEP 2015, DOI: 10.1002/ccr3.400

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    Gaucher disease: pathological mechanisms and modern management

    British Journal of Haematology

    Volume 129, Issue 2, April 2005, Pages: 178–188, Marina Jmoudiak and Anthony H. Futerman

    Version of Record online : 6 APR 2005, DOI: 10.1111/j.1365-2141.2004.05351.x

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    New insights into therapeutic options for Pompe disease

    IUBMB Life

    Volume 63, Issue 11, November 2011, Pages: 979–986, Emmanuel Richard, Gaëlle Douillard-Guilloux and Catherine Caillaud

    Version of Record online : 14 OCT 2011, DOI: 10.1002/iub.529

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    Toward the successful delivery of lysosomal enzymes across the blood–brain barrier

    Clinical and Experimental Neuroimmunology

    Volume 4, Issue 2, August 2013, Pages: 228–238, Akihiko Urayama

    Version of Record online : 27 AUG 2013, DOI: 10.1111/cen3.12037

  11. The role of immune tolerance induction in restoration of the efficacy of ERT in Pompe disease

    American Journal of Medical Genetics Part C: Seminars in Medical Genetics

    Volume 160C, Issue 1, 15 February 2012, Pages: 30–39, Emanuela Lacaná, Lynne P. Yao, Anne R. Pariser and Amy S. Rosenberg

    Version of Record online : 17 JAN 2012, DOI: 10.1002/ajmg.c.31316

  12. Subsurface architecture of the Boulder Creek Critical Zone Observatory from electrical resistivity tomography

    Earth Surface Processes and Landforms

    Volume 38, Issue 12, 30 September 2013, Pages: 1417–1431, Matthias Leopold, Jörg Völkel, Juliane Huber and David Dethier

    Version of Record online : 7 MAY 2013, DOI: 10.1002/esp.3420

  13. You have full text access to this OnlineOpen article
    Extracellular matrix components: An intricate network of possible biomarkers for lysosomal storage disorders?

    FEBS Letters

    Volume 587, Issue 8, April 17, 2013, Pages: 1258–1267, Spyros P. Batzios, Dimitrios I. Zafeiriou and Eleni Papakonstantinou

    Version of Record online : 26 FEB 2013, DOI: 10.1016/j.febslet.2013.02.035

  14. Expanding newborn screening for lysosomal disorders: Opportunities and challenges

    Developmental Disabilities Research Reviews

    Volume 17, Issue 1, 2011, Pages: 9–14, Darrel J. Waggoner and Christopher A. Tan

    Version of Record online : 23 MAR 2012, DOI: 10.1002/ddrr.132

  15. Reversibility of cellular and organ pathology in enzyme replacement trials in animal models of lysosomal storage diseases

    Acta Paediatrica

    Volume 95, Issue S451, April 2006, Pages: 93–99, Volkmar Gieselmann

    Version of Record online : 2 JAN 2007, DOI: 10.1111/j.1651-2227.2006.tb02397.x

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    Estrogen replacement therapy for menopausal women with a history of breast carcinoma


    Volume 95, Issue 9, 1 November 2002, Pages: 1817–1826, Rena Vassilopoulou-Sellin, Deborah S. Cohen, Gabriel N. Hortobagyi, Mary Jean Klein, Marsha McNeese, S. Eva Singletary, Terry L. Smith and Richard L. Theriault

    Version of Record online : 25 OCT 2002, DOI: 10.1002/cncr.10913

  17. You have free access to this content
    Imaging and characterization of solute transport during two tracer tests in a shallow aquifer using electrical resistivity tomography and multilevel groundwater samplers

    Water Resources Research

    Volume 46, Issue 3, March 2010, Kerstin Müller, Jan Vanderborght, Andreas Englert, Andreas Kemna, Johan A. Huisman, Joerg Rings and Harry Vereecken

    Version of Record online : 3 MAR 2010, DOI: 10.1029/2008WR007595

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    Quantitative imaging of solute transport in an unsaturated and undisturbed soil monolith with 3-D ERT and TDR

    Water Resources Research

    Volume 44, Issue 12, December 2008, Johannes Koestel, Andreas Kemna, Mathieu Javaux, Andrew Binley and Harry Vereecken

    Version of Record online : 10 DEC 2008, DOI: 10.1029/2007WR006755

  19. Effects of short-to-long term enzyme replacement therapy (ERT) on skeletal muscle tissue in late onset Pompe disease (LOPD)

    Neuropathology and Applied Neurobiology

    M. Ripolone, R. Violano, D. Ronchi, S. Mondello, A. Nascimbeni, I. Colombo, G. Fagiolari, A. Bordoni, F. Fortunato, V. Lucchini, S. Simona, M. Filosto, O. Musumeci, P. Tonin, T. Mongini, S. Previtali, L. Morandi, C. Angelini, M. Mora, M. Sandri, M. Sciacco, A. Toscano, G. P. Comi and M. Moggio

    Version of Record online : 4 JUL 2017, DOI: 10.1111/nan.12414

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    Saline tracer visualized with three-dimensional electrical resistivity tomography: Field-scale spatial moment analysis

    Water Resources Research

    Volume 41, Issue 5, May 2005, Kamini Singha and Steven M. Gorelick

    Version of Record online : 24 MAY 2005, DOI: 10.1029/2004WR003460