Publications by authors named "Atef Zaher"

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Clinical modeling of motor function to predict treatment efficacy and enable in silico treatment comparisons in infantile-onset Pompe disease.
Fatiha Rachedi, Rana Jreich, Susan Sparks, Atef Zaher, Kristina An Haack

CPT Pharmacometrics Syst Pharmacol· November 2025

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Quantitative Systems Pharmacology-Based Digital Twins Approach Supplements Clinical Trial Data for Enzyme Replacement Therapies in Pompe Disease.
Chanchala Kaddi, Mengdi Tao, Silke Bergeler, Kelly George, Hugo Geerts, Atef Zaher

Clin Pharmacol Ther· February 2025

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Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study.
Patrick B Deegan, Ozlem Goker-Alpan, Tarekegn Geberhiwot, Robert J Hopkin, Elena Lukina, Atef Zaher

Mol Genet Metab· February 2023

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Safety and efficacy of avalglucosidase alfa in individuals with infantile-onset Pompe disease enrolled in the phase 2, open-label Mini-COMET study: The 6-month primary analysis report.
Priya S Kishnani, David Kronn, Anaïs Brassier, Alexander Broomfield, James Davison, Atef Zaher

Genet Med· February 2023

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A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results.
Melissa Wasserstein, Robin Lachmann, Carla Hollak, Laila Arash-Kaps, Antonio Barbato, Atef Zaher

Genet Med· July 2022

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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, George A Diaz, Robin H Lachmann, Thomas Schiano, Melissa P Wasserstein, Atef Zaher

Mol Genet Metab· June 2021

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Expression of AR-V7 and ARv in Circulating Tumor Cells Correlates with Outcomes to Taxane Therapy in Men with Metastatic Prostate Cancer Treated in TAXYNERGY.
Scott T Tagawa, Emmanuel S Antonarakis, Ada Gjyrezi, Giuseppe Galletti, Seaho Kim, Atef Zaher

Clin Cancer Res· March 2019

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Randomized, Noncomparative, Phase II Trial of Early Switch From Docetaxel to Cabazitaxel or Vice Versa, With Integrated Biomarker Analysis, in Men With Chemotherapy-Naïve, Metastatic, Castration-Resistant Prostate Cancer.
Emmanuel S Antonarakis, Scott T Tagawa, Giuseppe Galletti, Daniel Worroll, Karla Ballman, Atef Zaher

J Clin Oncol· October 2017

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