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Hung Do

Amicus Therapeutics

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Publication

Featured researches published by Hung Do.

Molecular Genetics and Metabolism | 2008

114. Pharmacological chaperone therapy for Gaucher disease: Mechanism of action, a survey of responsive mutations and phase I clinical trial results

Brandon Wustman; Corey W. Pine; Brian Ranes; John J. Flanagan; David Palling; Hung Do; Frank Insinga; Gregory A. Grabowski; Neal J. Weinreb; Gregory M. Pastores; Paul M. Fernhoff; David J. Lockhart

Blood | 2007

Pharmacological Chaperone Therapy for the Treatment of Gaucher Disease: Results of Phase 1 Clinical Trials and a Clinical Ex Vivo Response Study with a Survey of Blood Markers for 53 Gaucher Patients.

Neal J. Weinreb; Gregory A. Grabowski; Gregory Pastores; Paul M. Fernhoff; David Palling; Corey W. Pine; B.E. Raines; F. Insinga; David J. Lockhart; Hung Do; Brandon Wustman

Neuromuscular Disorders | 2018

METABOLIC MYOPATHIES II

Yi Lun; Su Xu; Rebecca Soska; Anju Nair; Michelle Frascella; Anadina Garcia; A. Ponery; Jessie Feng; C. Della Valle; Russell Gotschall; Hung Do; Kenneth J. Valenzano; Richie Khanna

Molecular Genetics and Metabolism | 2018

A next-generation Fabry enzyme replacement therapy: A proprietary human α-galactosidase A co-formulated with a pharmacological chaperone, AT1001, shows greater substrate reduction than standard of care in Fabry mice

Su Xu; Adriane Schilling; Nestor Gomez; Michelle Frascella; Anandina Garcia; Rick L. Hamler; Daniel Ellsworth; Rebecca Soska; Anju Nair; Maria Cecilia Della Valle; Jessie Feng; Hayley Manger; Kenneth J. Valenzano; Hung Do; Russell Gotschall; Richie Khanna

Molecular Genetics and Metabolism | 2018

A major advance in the search for more effective therapy for Pompe disease

Nina Raben; Evelyn Ralston; Yi Lun; Abdul S. Ponery; Michelle Frascella; Anadina Garcia; Kenneth J. Valenzano; Russel Gotschall; Hung Do; Richie Khanna; Rosa Puertollano

Neuromuscular Disorders | 2017

P.217 - Stabilized next-generation recombinant human acid alpha-glucosidase ATB200 clears accumulated glycogen and reverses cellular dysfunction to increase muscle strength in a mouse model of Pompe disease

Su Xu; Yi Lun; Anju Nair; Michelle Frascella; Anadina Garcia; Rebecca Soska; A. Ponery; Adriane Schilling; C. Della Valle; Jessie Feng; Russell Gotschall; Hung Do; Kenneth J. Valenzano; Richie Khanna

Molecular Genetics and Metabolism | 2017

Stabilized next-generation recombinant human acid alpha-glucosidase ATB200 clears accumulated glycogen and reverses cellular dysfunction to increase functional muscle strength in a mouse model of Pompe disease

Hung Do; Yi Lun; Su Xu; Rebecca Soska; Anju Nair; Michelle Frascella; Anadina Garcia; Adriane Schilling; Cecilia Della Valle; Jessie Feng; Russell Gotschall; Kenneth J. Valenzano; Richie Khanna

Molecular Genetics and Metabolism | 2017

A novel recombinant human acid alpha-glucosidase, ATB200, leads to greater substrate reduction and improvement in Pompe disease-relevant markers compared to alglucosidase alfa in GAA KO mice

Yi Lun; Su Xu; Rebecca Soska; Anju Nair; Michelle Frascella; Anadina Garcia; Russell Gotschall; Hung Do; Kenneth J. Valenzano; Richie Khanna

Molecular Genetics and Metabolism | 2016

Co-administration of the pharmacological chaperone AT2221 with a proprietary recombinant human acid alfa-glucosidase leads to greater plasma exposure and substrate reduction compared to alglucosidase alfa

Richie Khanna; Su Xu; Deborah Hilliard; Yi Lun; Adriane Schilling; Rebecca Soska; Anju Nair; Kate Chang; Jessie Feng; Michelle Frascella; Anadina Garcia; Kimberly Pendino; Franklin K. Johnson; Elfrida R. Benjamin; Russell Gotschall; Hung Do; Kenneth J. Valenzano

Molecular Genetics and Metabolism | 2015

Novel recombinant human acid α-glucosidase with optimal glycosylation is significantly better than standard of care enzyme replacement for glycogen clearance in skeletal muscles of GAA knock-out mice

Russell Gotschall; Su Xu; Yi Lun; Adriane Schilling; Rebecca Soska; Michelle Frascella; Anadina Garcia; Jessie Feng; Kate Chang; Nastry Brignol; Richie Khanna; Kenneth J. Valenzano; Hung Do

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Collaboration

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Gregory A. Grabowski

Cincinnati Children's Hospital Medical Center

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Neal J. Weinreb

University of Miami

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Paul M. Fernhoff

Emory University

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Gregory Pastores

City University of New York

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Nina Raben

National Institutes of Health

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Rick L. Hamler

University of Michigan

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Rosa Puertollano

National Institutes of Health

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Gregory M. Pastores

Mater Misericordiae University Hospital

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