Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease:

Slides:



Advertisements
Similar presentations
2015 Alzheimer's disease facts and figures Alzheimer's & Dementia: The Journal of the Alzheimer's Association Volume 11, Issue 3, Pages (March.
Advertisements

Carbohydrate and fat utilization during rest and physical activity Katarina Melzer European e-Journal of Clinical Nutrition and Metabolism Volume 6, Issue.
EASL Clinical Practice Guidelines: Vascular diseases of the liver Journal of Hepatology Volume 64, Issue 1, Pages (January 2016) DOI: /j.jhep
Celiac Disease Genetics: Current Concepts and Practical Applications Ludvig M. Sollid, Benedicte A. Lie Clinical Gastroenterology and Hepatology Volume.
Volume 133, Issue 3, Pages (March 2014)
Does statin therapy reduce plasma VEGF levels in humans
Relationship between high normal TSH levels and metabolic syndrome components in type 2 diabetic subjects with euthyroidism  Lilit Petrosyan  Journal.
Clinical evaluation of recombinant human platelet – derived growth factor for the treatment of lower extremity diabetic ulcers  David L. Steed, MD, the.
The PSA Era is not Over for Prostate Cancer
Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment—A retrospective.
Adjunctive therapy with interferon-gamma for the treatment of pulmonary tuberculosis: a systematic review  X.-F. Gao, Z.-W. Yang, J. Li  International.
Use of Low-Molecular-Weight Heparin in the Treatment of Venous Thromboembolic Disease: Answers to Frequently Asked Questions  Scott C. Litin, M.D., John.
Rapid virological response tailors the duration of treatment in hepatitis C virus genotype 3 patients treated with pegylated interferon alfa-2a and ribavirin.
A practical fluorometric assay method to measure lysosomal acid lipase activity in dried blood spots for the screening of cholesteryl ester storage disease.
Effect of dornase alfa on inflammation and lung function: Potential role in the early treatment of cystic fibrosis  Michael W. Konstan, Felix Ratjen 
C.-S. Lee, J.-H. Lee  Clinical Microbiology and Infection 
L. S. Tzouvelekis, A. Markogiannakis, E. Piperaki, M. Souli, G. L
L. Boyanova  Clinical Microbiology and Infection 
P. Melin  Clinical Microbiology and Infection 
Alzheimer's disease drug development pipeline: 2018
American Journal of Kidney Diseases 
Volume 44, Issue 4, Pages (April 2006)
Low platelet counts predict mortality in severe community acquired pneumonia in children under 5 years of age: A hospital based observational study  Anubha.
Clinical Microbiology and Infection
C.-S. Lee, J.-H. Lee  Clinical Microbiology and Infection 
Virological tools to diagnose and monitor hepatitis C virus infection
Association between particular polymorphic residues on apical membrane antigen 1 (AMA-1) and platelet levels in patients with vivax malaria  P. Grynberg,
Advances in the Therapy of Prostate Cancer–Induced Bone Disease: Current Insights and Future Perspectives on the RANK/RANKL Pathways  Kurt Miller, Arnulf.
The PSA Era is not Over for Prostate Cancer
K. L. Nash, M. E. Allison, D. McKeon, D. J. Lomas, C. S. Haworth, D
Pain in cancer survivors; filling in the gaps
Bjorn van Anrooij, MD, Joanne N. G. Oude Elberink, MD, PhD, Lambert F
V.E. Nambudiri, R.C. Dwyer, C.A. Camargo, T.S. Kupper, D.J. Pallin 
Patient-Reported Pressure Ulcer Pain: A Mixed-Methods Systematic Review  Claudia Gorecki, MSc, S. José Closs, PhD, RGN, Jane Nixon, PhD, RN, Michelle Briggs,
Clinical Microbiology and Infection
Effect of Omalizumab on Blood Basophil Counts in Patients with Chronic Idiopathic/Spontaneous Urticaria  Sarbjit S. Saini, Theodore A. Omachi, Benjamin.
Assessment of nephrotoxicity in patients receiving amphotericin B lipid complex: a pharmacosurveillance study in Spain  J.-M. Aguado, C. Lumbreras, D.
Effects of Ruxolitinib Treatment on Metabolic and Nutritional Parameters in Patients With Myelofibrosis From COMFORT-I  Ruben A. Mesa, Srdan Verstovsek,
Modifications of haematological series in patients co-infected with human immunodeficiency virus and hepatitis C virus during treatment with interferon.
Volume 26, Issue 2, Pages (February 2018)
CMI editorial report 2011 Clinical Microbiology and Infection
C. Fabrizio, A. Procopio  Clinical Microbiology and Infection 
Retrospective analysis of the incidence of epidural haematoma in patients with epidural catheters and abnormal coagulation parameters  P. Gulur, B. Tsui,
M. Or, M. Samish, T. Waner, S. Harrus 
Volume 119, Issue 6, Pages (December 2000)
Does motor block related to long-acting brachial plexus block cause patient dissatisfaction after minor wrist and hand surgery? A randomized observer-blinded.
Gender differences in treatment outcomes of tuberculosis patients in Taiwan: a prospective observational study  J.-Y. Feng, S.-F. Huang, W.-Y. Ting, Y.-C.
Challenges in design and interpretation of chronic pain trials
S. David, M. Mentasti, J. Parkhill, V.J. Chalker 
Recommendations for the management of patients with homozygous familial hypercholesterolaemia: Overview of a new European Atherosclerosis Society consensus.
Interleukin-8 production by polymorphonuclear leukocytes from patients with chronic infected leg ulcers treated with Lactobacillus plantarum  M.C. Peral,
Current experience in treating invasive zygomycosis with posaconazole
An informal discussion among emergency nurses about their current clinical practice: What’s new and what works  Deborah Blazys, RN, BSN  Journal of Emergency.
Efficacy and Safety of a Single Dose versus a Multiple Dose Regimen of Mebendazole against Hookworm Infections in Children: A Randomised, Double-blind.
Molecular identification of Acinetobacter sp
Volume 17, Issue 2, Pages (February 1950)
L. R. Ásmundsdóttir, H. Erlendsdóttir, A. L. Gísladóttir, M
Statin use and clinical outcomes among pneumonia patients
What is an “eosinophilic phenotype” of asthma?
Do old pharmacokinetic parameter estimates predict new data?
Increased sputum endotoxin levels are associated with an impaired lung function response to oral steroids in asthmatic patients  Charles McSharry, PhD,
Exercise-induced bronchoconstriction in children: Montelukast attenuates the immediate-phase and late-phase responses  Raul E. Melo, MD, Dirceu Solé,
Use of Low-Molecular-Weight Heparin in the Treatment of Venous Thromboembolic Disease: Answers to Frequently Asked Questions  Scott C. Litin, M.D., John.
Olga Vornicova, MD, Gil Bar-Sela, MD  JAAD Case Reports 
GlideScope® video laryngoscope: a randomized clinical trial in 203 paediatric patients†   J.-T. Kim, H.-S. Na, J.-Y. Bae, D.-W. Kim, H.-S. Kim, C.S. Kim,
Rapid onset of action in patients with moderate-to-severe psoriasis treated with brodalumab: A pooled analysis of data from two phase 3 randomized clinical.
Scott K. Fung, Anna S.F. Lok  Clinical Gastroenterology and Hepatology 
Impact of antibiotic restrictions: the patient's perspective
Footnotes1 Journal of Allergy and Clinical Immunology
Impact of vitamin D on infectious disease-tuberculosis-a review
Presentation transcript:

Reductions in glucosylsphingosine (lyso-Gb1) in treatment-naïve and previously treated patients receiving velaglucerase alfa for type 1 Gaucher disease: Data from phase 3 clinical trials  Deborah Elstein, Björn Mellgard, Quinn Dinh, Lan Lan, Yongchang Qiu, Claudia Cozma, Sabrina Eichler, Tobias Böttcher, Ari Zimran  Molecular Genetics and Metabolism   Volume 122, Issue 1, Pages 113-120 (September 2017) DOI: 10.1016/j.ymgme.2017.08.005 Copyright © 2017 The Authors Terms and Conditions

Fig. 1 Overview of clinical trials. EOW, every other week. Molecular Genetics and Metabolism  2017 122, 113-120DOI: (10.1016/j.ymgme.2017.08.005) Copyright © 2017 The Authors Terms and Conditions

Fig. 2 Change in lyso-Gb1 concentrations during velaglucerase alfa treatment. (A) Treatment-naïve patients: all patients (n=22). (B) Treatment-naïve patients: percentage changes. (C) Switch patients: all patients (n=21). (D) Switch patients: percentage changes. Mean values are indicated by closed squares, median values are indicated by open circles. Molecular Genetics and Metabolism  2017 122, 113-120DOI: (10.1016/j.ymgme.2017.08.005) Copyright © 2017 The Authors Terms and Conditions

Fig. 3 Impact of genotype on lyso-Gb1 concentrations and response during velaglucerase alfa treatment. (A) Treatment-naïve patients: observed concentrations. (B) Treatment-naïve patients: percentage changes. (C) Switch patients: observed concentrations. (D) Switch patients: percentage changes. Mean values are indicated by closed squares, median values are indicated by open circles. Molecular Genetics and Metabolism  2017 122, 113-120DOI: (10.1016/j.ymgme.2017.08.005) Copyright © 2017 The Authors Terms and Conditions

Fig. 4 Spearman correlations between lyso-Gb1 concentrations and clinical disease parameters during velaglucerase alfa treatment. (A) treatment-naïve patients: platelet counts. (B) Treatment-naïve patients: spleen volume. (C) Switch patients: platelet counts. (D) Switch patients: spleen volume. MN, multiples of normal. Molecular Genetics and Metabolism  2017 122, 113-120DOI: (10.1016/j.ymgme.2017.08.005) Copyright © 2017 The Authors Terms and Conditions

Feedback: Privacy Policy Feedback
Do Not Sell My Personal Information
About project: SlidePlayer Terms of Service

© 2025 SlidePlayer.com. Inc. All rights reserved.