Beth L. Thurberg

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Articole 50

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2020-06-24

Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment

Beth L. Thurberg, Melissa P Wasserstein
2019-12-10

Improved muscle function in a phase I/II clinical trial of albuterol in Pompe disease

Beth L. Thurberg
2019-04-03

Low-dose agalsidase beta treatment in male pediatric patients with Fabry disease: A 5-year randomized controlled trial

Lorne Clarke, Anna Tylki-Szymańska, Beth L. Thurberg
2018-07-05

Correction of Biochemical Abnormalities and Improved Muscle Function in a Phase I/II Clinical Trial of Clenbuterol in Pompe Disease

Beth L. Thurberg
2018-12-17

Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study

Kerry E. Culm-Merdek, Beth L. Thurberg
2017-03-25

Chronic intestinal pseudo-obstruction. Did you search for lysosomal storage diseases?

Beth L. Thurberg
2016-10-01

Fabry disease: Four case reports of meningioma and a review of the literature on other malignancies

Dominique P Germain, Beth L. Thurberg
2016-01-01

Natural Progression of Canine Glycogen Storage Disease Type IIIa

Beth L. Thurberg
2016-06-23

Clearance of Hepatic Sphingomyelin by Olipudase Alfa Is Associated With Improvement in Lipid Profiles in Acid Sphingomyelinase Deficiency

Simon Jones, Beth L. Thurberg, Melissa P Wasserstein
2016-11-10

Systemic Correction of Murine Glycogen Storage Disease Type IV by an AAV-Mediated Gene Therapy.

Elizabeth D Brooks, Beth L. Thurberg
2016-05-19

Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study

Benedikt Schoser, Volker Straub, Beth L. Thurberg
2015-09-03

Gastrointestinal involvement in Fabry disease. So important, yet often neglected

Beth L. Thurberg
2015-05-08

Characterization of early disease status in treatment-naive male paediatric patients with Fabry disease enrolled in a randomized clinical trial

Daniel-G. Bichet, Lorne Clarke, Anna Tylki-Szymańska, Beth L. Thurberg
2015-05-30

Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency

Kerry E. Culm-Merdek, Beth L. Thurberg, Melissa P Wasserstein
2015-04-02

Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann-Pick disease type B (acid sphingomyelinase deficiency).

Beth L. Thurberg, Melissa P Wasserstein, Margaret M. McGovern
2015-01-01

A Phase 4 Prospective Study in Patients with Adult Pompe Disease Treated with Alglucosidase Alfa

Benedikt Schoser, Volker Straub, Beth L. Thurberg
2015-03

α-Galactosidase A knockout mice: progressive organ pathology resembles the type 2 later-onset phenotype of Fabry disease

John Marshall, John Lydon, Beth L. Thurberg, Robert J. Desnick
2014-02-08

Correction of glycogen storage disease type III with rapamycin in a canine model.

Haiqing Yi, Elizabeth D Brooks, Beth L. Thurberg
2014-07-14

Prominent vacuolation of the eyelid levator muscle in an early-treated child with infantile-onset Pompe disease

Yin-Hsiu Chien, Ni-Chung Lee, Wuh-liang Hwu, Beth L. Thurberg, Fuu-Jen Tsai
2014-07-12

Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency:the utility of animal models of disease in the toxicological evaluation of potential therapeutics

Beth L. Thurberg
2013-08-14

Myostatin and insulin-like growth factor I: potential therapeutic biomarkers for pompe disease

Der-Sheng Han, Wei-shiung Yang, Yin-Hsiu Chien, Wuh-liang Hwu, Beth L. Thurberg
2012-08-01

Liver and skin histopathology in adults with acid sphingomyelinase deficiency (Niemann-Pick disease type B)

Beth L. Thurberg, Melissa P Wasserstein, Margaret M. McGovern
2012-06-26

Characterization of a canine model of glycogen storage disease type IIIa

Beth L. Thurberg, Priya S Kishnani
2012-09-19

Adjunctive β2-agonists reverse neuromuscular involvement in murine Pompe disease

Beth L. Thurberg
2012-05-18

Autopsy findings in late-onset Pompe disease: a case report and systematic review of the literature

Beth L. Thurberg
2012-12-27

Alglucosidase alfa enzyme replacement therapy as a therapeutic approach for glycogen storage disease type III.

Adviye A Tolun, William E. Kraus, Yuan-Tsong Chen, Beth L. Thurberg
2012-03-01

Early pathologic changes and responses to treatment in patients with later-onset Pompe disease

Yin-Hsiu Chien, Pei-Hsin Huang, Wuh-liang Hwu, Beth L. Thurberg, Wang-Tso Lee
2011-11-11

β2 Agonists enhance the efficacy of simultaneous enzyme replacement therapy in murine Pompe disease

Beth L. Thurberg, Priya S Kishnani
2011-11-10

Histologic abnormalities of placental tissues in Fabry disease: a case report and review of the literature

Beth L. Thurberg
2011-05-05

Expanding the clinical spectrum of late-onset Pompe disease: dilated arteriopathy involving the thoracic aorta, a novel vascular phenotype uncovered.

Beth L. Thurberg
2011-01-13

Later-onset Pompe disease: early detection and early treatment initiation enabled by newborn screening

Wuh-liang Hwu, Yin-Hsiu Chien, Ni-Chung Lee, Beth L. Thurberg, Fuu-Jen Tsai
2010-10-22

Antibody formation and mannose-6-phosphate receptor expression impact the efficacy of muscle-specific transgene expression in murine Pompe disease

Haiqing Yi, Beth L. Thurberg

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