Showing 1 - 8 results of 8 for search 'Raymond Y. Wang', query time: 0.03s Refine Results
  1. 1
    An updated management approach of Pompe disease patients with high-sustained anti-rhGAA IgG antibody titers: experience with bortezomib-based immunomodulation

    An updated management approach of Pompe disease patients with high-sustained anti-rhGAA IgG antibody titers: experience with bortezomib-based immunomodulation by Ankit K. Desai, Garima Shrivastava, Christina L. Grant, Raymond Y. Wang, Raymond Y. Wang, Trevor D. Burt, Trevor D. Burt, Priya S. Kishnani

    Published 2024-03-01
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  2. 2
    Two-year follow-up of gait and postural control following initiation of recombinant human tripeptidyl intracerebroventricular enzyme replacement therapy in two atypical CLN2 patients

    Two-year follow-up of gait and postural control following initiation of recombinant human tripeptidyl intracerebroventricular enzyme replacement therapy in two atypical CLN2 patien... by Rahul Soangra, Marybeth Grant-Beuttler, Harriet Chang, Raymond Y. Wang

    Published 2025-01-01
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  3. 3
    Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells

    Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells by Chloe L. Christensen, Shih-Hsin Kan, Perla Andrade-Heckman, Allisandra K. Rha, Jerry F. Harb, Raymond Y. Wang

    Published 2024-06-01
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  4. 4
    Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing

    Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing by Allisandra K. Rha, Chloe L. Christensen, Shih-Hsin Kan, Jerry F. Harb, Perla Andrade-Heckman, Raymond Y. Wang

    Published 2024-12-01
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  5. 5
    Growth patterns in patients with mucopolysaccharidosis VII

    Growth patterns in patients with mucopolysaccharidosis VII by Adriana M. Montaño, Agnieszka Różdżyńska-Świątkowska, Agnieszka Jurecka, Antonio Nino Ramirez, Lin Zhang, Deborah Marsden, Raymond Y. Wang, Paul Harmatz

    Published 2023-09-01
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  6. 6
    Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice

    Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice by Caitlin C. Calhoun, Shih-Hsin Kan, Alexander E. Stover, Jerry F. Harb, Edwin S. Monuki, Raymond Y. Wang, Philip H. Schwartz

    Published 2024-12-01
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  7. 7
    CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease

    CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease by Shih-hsin Kan, Jeffrey Y. Huang, Jerry Harb, Allisandra Rha, Nancy D. Dalton, Chloe Christensen, Yunghang Chan, Jeremy Davis-Turak, Jonathan Neumann, Raymond Y. Wang

    Published 2022-12-01
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  8. 8
    Base editing corrects the common Salla disease SLC17A5 c.115C>T variant

    Base editing corrects the common Salla disease SLC17A5 c.115C>T variant by Jerry F. Harb, Chloe L. Christensen, Shih-Hsin Kan, Allisandra K. Rha, Perla Andrade-Heckman, Laura Pollard, Richard Steet, Jeffrey Y. Huang, Raymond Y. Wang

    Published 2023-12-01
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