Ann Barbier

Translate Bio (United States) — United States

Immunology and Microbiology · Life Sciences

31h-index3.6kcitations125works0.02yr avg
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Research Topics

Mast cells and histamine(20), Receptor Mechanisms and Signaling(16), Lysosomal Storage Disorders Research(13), Glycogen Storage Diseases and Myoclonus(9), Olfactory and Sensory Function Studies(8)

Publications125 total

Inhaled mRNA therapy for treatment of cystic fibrosis: Interim results of a randomized, double‐blind, placebo‐controlled phase 1/2 clinical study
Journal of Cystic Fibrosis·2023· 119 citedOpen Access
The clinical progress of mRNA vaccines and immunotherapies
Nature Biotechnology·2022· 710 citedOpen Access
544: Safety and tolerability of single and repeat doses of MRT5005, an inhaled CFTR mRNA replacement therapy, in adult CF patients
Journal of Cystic Fibrosis·2021· 2 citedOpen Access
Distribution of cystic fibrosis patients not eligible to studied CFTR modulators in Europe
Archivio Istituzionale della Ricerca (Universita Degli Studi Di Milano)·2020Open Access
WS13.4 Distribution of cystic fibrosis patients not eligible to studied CFTR modulators in Europe
Journal of Cystic Fibrosis·2020Open Access
A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II
UNC Libraries·2020Open Access
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency
New England Journal of Medicine·2019· 154 citedOpen Access
Phase 1 Single‐ and Multiple‐Ascending‐Dose Randomized Studies of the Safety, Pharmacokinetics, and Pharmacodynamics of AG‐348, a First‐in‐Class Allosteric Activator of Pyruvate Kinase R, in Healthy Volunteers
Clinical Pharmacology in Drug Development·2018· 51 cited
Results Update from the DRIVE PK Study: Effects of AG-348, a Pyruvate Kinase Activator, in Patients with Pyruvate Kinase Deficiency
Blood·2017· 2 cited
Population Pharmacokinetics and Pharmacodynamics of AG-519, a Pyruvate Kinase Activator for the Treatment of Pyruvate Kinase Deficiency, in Human Healthy Volunteers
Blood·2016
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Doses of AG-519, an Allosteric Activator of Pyruvate Kinase-R, in Healthy Subjects
Blood·2016
Effects of AG-348, a Pyruvate Kinase Activator, on Anemia and Hemolysis in Patients with Pyruvate Kinase Deficiency: Data from the DRIVE PK Study
Blood·2016· 6 cited
Characterization of Metabolic Response to AG-348, an Allosteric Activator of Red Cell Pyruvate Kinase, in Healthy Volunteers and Pyruvate Kinase Deficiency Patients
Blood·2016· 1 cited
Patient Voice in Rare Disease Drug Development and Endpoints
Therapeutic Innovation & Regulatory Science·2016· 22 cited
A phase 1/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA
Molecular Genetics and Metabolism·2016· 124 citedOpen Access
Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions
Molecular Genetics and Metabolism·2016· 31 citedOpen Access
A Novel LC–MS/MS Assay for Heparan Sulfate Screening in The Cerebrospinal Fluid of Mucopolysaccharidosis Iiia Patients
Bioanalysis·2016· 24 cited
Interférences par une IgM monoclonale dans un bilan biochimique : détection et recommandations Interferences by a monoclonal IgM in biochemical analyses: detection and recommendations
2016
DRIVE PK: A Phase 2 Trial of AG-348 in Patients with Pyruvate Kinase Deficiency
Blood·2015· 1 cited
Levels of glycosaminoglycans in the cerebrospinal fluid of healthy young adults, surrogate-normal children, and Hunter syndrome patients with and without cognitive impairment
Molecular Genetics and Metabolism Reports·2015· 29 citedOpen Access
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Frequent Co-authors

Nicholas I. Carruthers(18), Sandy J. Wilson(17), Timothy W. Lovenberg(16), Jamin D. Boggs(13), Brian Lord(10), Michael A. Letavic(9), Leah Aluisio(7), Richard L. Apodaca(6), John M. Keith(6), Pascal Bonaventure(6), Wei Xiao(5), Tarun B. Patel(5), Kirsten L. Miller(5), Christine Dugovic(4), Luying Pan(4), Curt Mazur(4), Romain A. Lefebvre(4), Lisa Dvorak(4), Arian Pano(4), Ian Fraser(4)