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Biomarkers in Pharmaceutical Research

Clinical chemistry (Baltimore, Md.), 2015-11, Vol.61 (11), p.1343-1353

2015

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Cerebrospinal fluid biomarkers distinguish postmortem-confirmed Alzheimer's disease from other dementias and healthy controls in the OPTIMA cohort

Journal of Alzheimer's disease, 2015-01, Vol.44 (2), p.525-539

2015

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Identification of Novel Brain Biomarkers

Clinical chemistry (Baltimore, Md.), 2006-09, Vol.52 (9), p.1713-1721

2006

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The Brain Injury Biomarker VLP-1 Is Increased in the Cerebrospinal Fluid of Alzheimer Disease Patients

Clinical chemistry (Baltimore, Md.), 2008-10, Vol.54 (10), p.1617-1623

2008

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Fabry Disease Patient-Reported Outcome (FD-PRO) demonstrates robust measurement properties for assessing symptom severity in Fabry disease

Molecular genetics and metabolism reports, 2021-12, Vol.29, p.100824-100824, Article 100824

2021

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Establishment and validation of analytical reference panels for the standardization of quantitative BCR-ABL1 measurements on the international scale

Clinical chemistry (Baltimore, Md.), 2013-06, Vol.59 (6), p.938-948

2013

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Glycosphingolipid metabolism and polycystic kidney disease

Cellular signalling, 2020-05, Vol.69, p.109526-109526, Article 109526

2020

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Investigational Therapy of Alport Syndrome With ELX-02: FR-PO322

Journal of the American Society of Nephrology, 2022-11, Vol.33 (11S), p.412-413

2022

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SaO002QUANTITATIVE RELATIONSHIP BETWEEN TKV GROWTH RATE AND eGFR RATE OF DECLINE IN ADPKD PATIENTS

Nephrology, dialysis, transplantation, 2019-06, Vol.34 (Supplement_1)

2019

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MO035HISTORICAL CONTROL ANALYSIS DEMONSTRATES SUPERIOR REDUCTION OF PLASMA GLOBOTRIAOSYLCERAMIDE BY VENGLUSTAT COMPARED WITH PLACEBO OR AGALSIDASE BETA IN CLASSIC FABRY DISEASE PATIENTS

Nephrology, dialysis, transplantation, 2020-06, Vol.35 (Supplement_3)

2020

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Glucosylceramide Synthase Inhibition with Venglustat in Classic Fabry Disease Patients Leads to Progressive Reduction of Endothelial Cell Globotriaosylceramide Inclusion Volume: PO1593

Journal of the American Society of Nephrology, 2020-10, Vol.31 (10S), p.506-507

2020

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Novel read through agent: ZKN-0013 demonstrates efficacy in APCmin model of familial adenomatous polyposis

Journal of molecular medicine (Berlin, Germany), 2023-04, Vol.101 (4), p.375-385

2023

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Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial

American journal of kidney diseases, 2023-05, Vol.81 (5), p.517-527.e1

2023

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P0043SYMPTOMS IN PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE: FINDINGS FROM QUALITATIVE PATIENT INTERVIEWS

Nephrology, dialysis, transplantation, 2020-06, Vol.35 (Supplement_3)

2020

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FGFR and FGF ligand overexpression in lung cancer: Implications for targeted therapy

Journal of clinical oncology, 2014-05, Vol.32 (15_suppl), p.11080-11080

2014

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Predictive multi-omics analysis of BPM31510 in combination with gemcitabine in a phase 2 study of advanced pancreatic ductal adenocarcinoma (PDAC)

Journal of clinical oncology, 2024-01, Vol.42 (3_suppl), p.696-696

2024

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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

Molecular genetics and metabolism, 2023-02, Vol.138 (2), p.106963-106963, Article 106963

2023

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Biomarkers of tissue injury

Biomarkers in medicine, 2008-02, Vol.2 (1), p.81-92

2008

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Correction of cilia structure and function alleviates multi-organ pathology in Bardet–Biedl syndrome mice

Human molecular genetics, 2020-08, Vol.29 (15), p.2508-2522

2020

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Abstract 6518: BPM 31510: Targeting the tumor microenvironment (TME) via mitochondrial-mediated ROS production

Cancer research (Chicago, Ill.), 2024-03, Vol.84 (6_Supplement), p.6518-6518

2024

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STAGED-PKD: An Enriched, Seamless, Two-Stage Study for Venglustat Assessment in ADPKD: PO1577

Journal of the American Society of Nephrology, 2020-10, Vol.31 (10S), p.502-502

2020

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STAGED-PKD: Patient Enrichment and Modeling-Driven Efficient ADPKD Trial Design: PO1578

Journal of the American Society of Nephrology, 2020-10, Vol.31 (10S), p.502-502

2020

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Abstract 1904: RNA expression based screening of ALK gene fusion from formalin fixed non-small cell lung cancer samples

Cancer research (Chicago, Ill.), 2014-10, Vol.74 (19_Supplement), p.1904-1904

2014

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Long-term prognostic significance of early molecular response to imatinib in newly diagnosed chronic myeloid leukemia: an analysis from the International Randomized Study of Interferon and STI571 (IRIS)

Biomarkers in Pharmaceutical Research

Cerebrospinal fluid biomarkers distinguish postmortem-confirmed Alzheimer's disease from other dementias and healthy controls in the OPTIMA cohort

High Resolution Discovery Proteomics Reveals Candidate Disease Progression Markers of Alzheimer's Disease in Human Cerebrospinal Fluid

Identification of Novel Brain Biomarkers

The Brain Injury Biomarker VLP-1 Is Increased in the Cerebrospinal Fluid of Alzheimer Disease Patients

Fabry Disease Patient-Reported Outcome (FD-PRO) demonstrates robust measurement properties for assessing symptom severity in Fabry disease

Establishment and validation of analytical reference panels for the standardization of quantitative BCR-ABL1 measurements on the international scale

Glycosphingolipid metabolism and polycystic kidney disease

Investigational Therapy of Alport Syndrome With ELX-02: FR-PO322

SaO002QUANTITATIVE RELATIONSHIP BETWEEN TKV GROWTH RATE AND eGFR RATE OF DECLINE IN ADPKD PATIENTS

MO035HISTORICAL CONTROL ANALYSIS DEMONSTRATES SUPERIOR REDUCTION OF PLASMA GLOBOTRIAOSYLCERAMIDE BY VENGLUSTAT COMPARED WITH PLACEBO OR AGALSIDASE BETA IN CLASSIC FABRY DISEASE PATIENTS

Glucosylceramide Synthase Inhibition with Venglustat in Classic Fabry Disease Patients Leads to Progressive Reduction of Endothelial Cell Globotriaosylceramide Inclusion Volume: PO1593

Novel read through agent: ZKN-0013 demonstrates efficacy in APCmin model of familial adenomatous polyposis

Venglustat, a Novel Glucosylceramide Synthase Inhibitor, in Patients at Risk of Rapidly Progressing ADPKD: Primary Results of a Double-Blind, Placebo-Controlled, Phase 2/3 Randomized Clinical Trial

P0043SYMPTOMS IN PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE: FINDINGS FROM QUALITATIVE PATIENT INTERVIEWS

FGFR and FGF ligand overexpression in lung cancer: Implications for targeted therapy

Predictive multi-omics analysis of BPM31510 in combination with gemcitabine in a phase 2 study of advanced pancreatic ductal adenocarcinoma (PDAC)

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

Biomarkers of tissue injury

Correction of cilia structure and function alleviates multi-organ pathology in Bardet–Biedl syndrome mice

Abstract 6518: BPM 31510: Targeting the tumor microenvironment (TME) via mitochondrial-mediated ROS production

STAGED-PKD: An Enriched, Seamless, Two-Stage Study for Venglustat Assessment in ADPKD: PO1577

STAGED-PKD: Patient Enrichment and Modeling-Driven Efficient ADPKD Trial Design: PO1578

Abstract 1904: RNA expression based screening of ALK gene fusion from formalin fixed non-small cell lung cancer samples

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