Suchergebnisse - "Young, Sarah"

PubPharm (18)

1

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

enthalten in: Genetics in medicine : official journal of the American College of Medical Genetics | 2020

von Khan, A. | Case, L. | Herbert, M. | +7

2

Adjunctive β2-agonist treatment reduces glycogen independently of receptor-mediated acid α-glucosidase uptake in the limb muscles of mice with Pompe disease

enthalten in: FASEB journal : official publication of the Federation of American Societies for Experimental Biology | 2014

von Farah, B. | Madden, L. | Li, S. | +6

3

Quantitative computed tomography for enzyme replacement therapy in Pompe disease

enthalten in: Brain & development | 2012

von Yonee, C. | Toyoshima, M. | Young, S. | +7

4

The emerging phenotype of long-term survivors with infantile Pompe disease

enthalten in: Genetics in medicine : official journal of the American College of Medical Genetics | 2012

von Prater, S. | Banugaria, S. | DeArmey, S. | +8

5

Assessing disease severity in Pompe disease : the roles of a urinary glucose tetrasaccharide biomarker and imaging techniques

enthalten in: American journal of medical genetics. Part C, Seminars in medical genetics | 2012

von Young, S. | Piraud, M. | Goldstein, J. | +7

6

Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease

enthalten in: Genetics in medicine : official journal of the American College of Medical Genetics | 2012

von Messinger, Y. | Mendelsohn, N. | Rhead, W. | +12

7

Atypical immunologic response in a patient with CRIM-negative Pompe disease

enthalten in: Molecular genetics and metabolism | 2011

von Abbott, M. | Prater, S. | Banugaria, S. | +4

8

Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease

enthalten in: Molecular therapy : the journal of the American Society of Gene Therapy | 2010

von Sun, B. | Kulis, M. | Young, S. | +9

9

Impaired clearance of accumulated lysosomal glycogen in advanced Pompe disease despite high-level vector-mediated transgene expression

enthalten in: The journal of gene medicine | 2009

von Sun, B. | Zhang, H. | Bird, A. | +3

10

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

enthalten in: Genetics in medicine : official journal of the American College of Medical Genetics | 2009

von Young, S. | Zhang, H. | Corzo, D. | +4