Zoekresultaten - Sarah P. Young

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  1. 1
    Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance

    Enhanced Response to Enzyme Replacement Therapy in Pompe Disease after the Induction of Immune Tolerance door Baodong Sun, Andrew Bird, Sarah P. Young, Priya S. Kishnani, Yuqi Chen, Dwight D. Koeberl

    Gepubliceerd in 2007
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  2. 2
    Long-term monitoring of patients with infantile-onset Pompe disease on enzyme replacement therapy using a urinary glucose tetrasaccharide biomarker

    Long-term monitoring of patients with infantile-onset Pompe disease on enzyme replacement therapy using a urinary glucose tetrasaccharide biomarker door Sarah P. Young, Haoyue Zhang, Deyanira Corzo, Beth L. Thurberg, Deeksha Bali, Priya S. Kishnani, David S. Millington

    Gepubliceerd in 2009
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  3. 3
    Circulating Bmp10 acts through endothelial Alk1 to mediate flow-dependent arterial quiescence

    Circulating Bmp10 acts through endothelial Alk1 to mediate flow-dependent arterial quiescence door Derek W. Laux, Sarah P. Young, James P. Donovan, Corrine Mansfield, Paul D. Upton, Beth L. Roman

    Gepubliceerd in 2013
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  4. 4
    Persistent elevations of alkaline phosphatase as an early indicator of GM1 gangliosidosis

    Persistent elevations of alkaline phosphatase as an early indicator of GM1 gangliosidosis door Iskren Menkovic, Monika Williams, Neelam Makhijani, Rui Wei, Sarah P. Young, Areeg El‐Gharbawy, Ashlee R. Stiles

    Gepubliceerd in 2025
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  5. 5
    Interaction between <i>alk1</i> and blood flow in the development of arteriovenous malformations

    Interaction between <i>alk1</i> and blood flow in the development of arteriovenous malformations door Paola Corti, Sarah P. Young, Chia‐Yuan Chen, Michael J. Patrick, Elizabeth R. Rochon, Kerem Pekkan, Beth L. Roman

    Gepubliceerd in 2011
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  6. 6
    Efficacy of an Adeno-associated Virus 8-Pseudotyped Vector in Glycogen Storage Disease Type II

    Efficacy of an Adeno-associated Virus 8-Pseudotyped Vector in Glycogen Storage Disease Type II door Baodong Sun, Haoyue Zhang, Luis M. Franco, Sarah P. Young, Ayn Schneider, Andrew Bird, Andrea Amalfitano, Y.-T. Chen, Dwight D. Koeberl

    Gepubliceerd in 2004
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  7. 7
    Evasion of Immune Responses to Introduced Human Acid α-Glucosidase by Liver-Restricted Expression in Glycogen Storage Disease Type II

    Evasion of Immune Responses to Introduced Human Acid α-Glucosidase by Liver-Restricted Expression in Glycogen Storage Disease Type II door Luis M. Franco, Baodong Sun, Xiaoyi Yang, Andrew Bird, Haoyue Zhang, Ayn Schneider, T. T. Brown, Sarah P. Young, Timothy M. Clay, Andrea Amalfitano, Y.T. Chen, Dwight D. Koeberl

    Gepubliceerd in 2005
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  8. 8
    Urinary Biomarkers of Oxidative Status in a Clinical Model of Oxidative Assault

    Urinary Biomarkers of Oxidative Status in a Clinical Model of Oxidative Assault door Dora Il’yasova, Ivan Spasojević, Frances Wang, Adviye Ayper Tolun, Karel Baše, Sarah P. Young, P. Kelly Marcom, Jeffrey R. Marks, Gabriel Mixon, Richard T. DiGiulio, David S. Millington

    Gepubliceerd in 2010
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  9. 9
    Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease

    Immunomodulatory Gene Therapy Prevents Antibody Formation and Lethal Hypersensitivity Reactions in Murine Pompe Disease door Baodong Sun, Michael D. Kulis, Sarah P. Young, Amy Hobeika, Songtao Li, Andrew Bird, Haoyue Zhang, Y Li, Timothy M. Clay, Wesley Burks, Priya S. Kishnani, Dwight D. Koeberl

    Gepubliceerd in 2009
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  10. 10
    Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy

    Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy door Baodong Sun, Sarah P. Young, Ping Li, Chunhui Di, T. T. Brown, Maja Z. Salva, Songtao Li, Andrew Bird, Zhen Yan, Richard L. Auten, Stephen D. Hauschka, Dwight D. Koeberl

    Gepubliceerd in 2008
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  11. 11
    Liver fibrosis during clinical ascertainment of glycogen storage disease type III: a need for improved and systematic monitoring

    Liver fibrosis during clinical ascertainment of glycogen storage disease type III: a need for improved and systematic monitoring door Carine A. Halaby, Sarah P. Young, Stephanie Austin, Ela Stefanescu, Deeksha Bali, Lani Clinton, Brian P. Smith, Surekha Pendyal, Jariya Upadia, Gary R. Schooler, Alisha Mavis, Priya S. Kishnani

    Gepubliceerd in 2019
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  12. 12
    Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature

    Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature door Aleena Khan, Laura E. Case, Mrudu Herbert, Stephanie DeArmey, Harrison N. Jones, Kelly D. Crisp, Kanecia O. Zimmerman, Mai K. ElMallah, Sarah P. Young, Priya S. Kishnani

    Gepubliceerd in 2020
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  13. 13
    The emerging phenotype of long-term survivors with infantile Pompe disease

    The emerging phenotype of long-term survivors with infantile Pompe disease door Sean N. Prater, Suhrad G. Banugaria, Stephanie DeArmey, Eleanor G. Botha, Erin M. Stege, Laura E. Case, Harrison N. Jones, Chanika Phornphutkul, Raymond Wang, Sarah P. Young, Priya S. Kishnani

    Gepubliceerd in 2012
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  14. 14
    Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants

    Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants door Priya S. Kishnani, Paula Goldenberg, Stephanie DeArmey, James H. Heller, Danny Benjamin, Sarah P. Young, Deeksha Bali, Sue Ann Smith, Jennifer S. Li, Hanna Mandel, Dwight D. Koeberl, Amy S. Rosenberg, Y-T Chen

    Gepubliceerd in 2009
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  15. 15
    Acetyl- <scp>l</scp> -carnitine deficiency in patients with major depressive disorder

    Acetyl- <scp>l</scp> -carnitine deficiency in patients with major depressive disorder door Carla Nasca, Benedetta Bigio, Francis S. Lee, Sarah P. Young, Marin Kautz, Ashly Albright, James Beasley, David S. Millington, Aleksander A. Mathé, James H. Kocsis, James W. Murrough, Bruce S. McEwen, Natalie Rasgon

    Gepubliceerd in 2018
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  16. 16
    Rare Disorders of Metabolism with Elevated Butyryl- and Isobutyryl-Carnitine Detected by Tandem Mass Spectrometry Newborn Screening

    Rare Disorders of Metabolism with Elevated Butyryl- and Isobutyryl-Carnitine Detected by Tandem Mass Spectrometry Newborn Screening door Dwight D. Koeberl, Sarah P. Young, Niels Gregersen, Jerry Vockley, Wendy E. Smith, Daniel K. Benjamin, Yan An, S. D. Weavil, Shu Chaing, Deeksha Bali, Marie McDonald, Priya S. Kishnani, Ying-Tsong Chen, David S. Millington

    Gepubliceerd in 2003
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  17. 17
    Glycocalyx Breakdown Is Associated With Severe Disease and Fatal Outcome in Plasmodium falciparum Malaria

    Glycocalyx Breakdown Is Associated With Severe Disease and Fatal Outcome in Plasmodium falciparum Malaria door Tsin Wen Yeo, J. Brice Weinberg, Daniel A. Lampah, Enny Kenangalem, Margaret A. Bush, Youwei Chen, Ric N. Price, Sarah P. Young, Hao Y. Zhang, David S. Millington, Donald L. Granger, Nicholas M. Anstey

    Gepubliceerd in 2019
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  18. 18
    Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

    Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease door Yoav H. Messinger, Nancy J. Mendelsohn, William J. Rhead, David Dimmock, Eli Hershkovitz, Michael Champion, Simon Jones, Rebecca Olson, Amy White, Cara Wells, Deeksha Bali, Laura E. Case, Sarah P. Young, Amy S. Rosenberg, Priya S. Kishnani

    Gepubliceerd in 2012
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  19. 19
    Evaluation of X-Linked Adrenoleukodystrophy Newborn Screening in North Carolina

    Evaluation of X-Linked Adrenoleukodystrophy Newborn Screening in North Carolina door Stacey Lee, Kristin Clinard, Sarah P. Young, Catherine Rehder, Zheng Fan, Ali S. Çalıkoğlu, Deeksha Bali, Donald B. Bailey, Lisa M. Gehtland, David S. Millington, Hari S. Patel, Sara E. Beckloff, Scott J. Zimmerman, Cynthia M. Powell, Jennifer Taylor

    Gepubliceerd in 2020
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  20. 20
    Mitochondrial NADP(H) deficiency due to a mutation in NADK2 causes dienoyl-CoA reductase deficiency with hyperlysinemia

    Mitochondrial NADP(H) deficiency due to a mutation in NADK2 causes dienoyl-CoA reductase deficiency with hyperlysinemia door Sander M. Houten, Simone Denis, Heleen te Brinke, Aldo Jongejan, Antoine H. C. van Kampen, Edward J. Bradley, Frank Baas, Raoul C. M. Hennekam, David S. Millington, Sarah P. Young, Dianne M. Frazier, Müge Güçsavaş‐Çalıkoğlu, Ronald J. A. Wanders

    Gepubliceerd in 2014
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