Abstract

This subproject is one of many research subprojects utilizing theresources provided by a Center grant funded by NIH/NCRR. The subproject andinvestigator (PI) may have received primary funding from another NIH source,and thus could be represented in other CRISP entries. The institution listed isfor the Center, which is not necessarily the institution for the investigator.This study is a multicenter, phase 2, open-label trial of small molecule chaperone therapy with AT1001 in patients with Fabry disease due to responding missense mutations. The trial consists of a 6-week-dose escalation phase followed by a 6-week treatment phase that may be extended an additional 36 weeks. Twenty male patients at 3 sites are planned to be enrolled to complete 12. The endpoints are safety, pharmacokinetic, and pharmacodynamic parameters.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000425-39
Application #
7717216
Study Section
Special Emphasis Panel (ZRR1-CR-5 (01))
Project Start
2007-12-01
Project End
2008-11-30
Budget Start
2007-12-01
Budget End
2008-11-30
Support Year
39
Fiscal Year
2008
Total Cost
$4,386
Indirect Cost

  Institution

Name
La Biomed Research Institute/ Harbor UCLA Medical Center
Department
Type
DUNS #
069926962
City
Torrance
State
CA
Country
United States
Zip Code
90502

  Publications

Sharma, Shilpa; Mehta, Puja K; Arsanjani, Reza et al. (2018) False-positive stress testing: Does endothelial vascular dysfunction contribute to ST-segment depression in women? A pilot study. Clin Cardiol 41:1044-1048
Shufelt, Chrisandra; Manson, Joann (2018) Managing Menopause by Combining Evidence With Clinical Judgment. Clin Obstet Gynecol 61:470-479
Cherukuri, Lavanya; Smith, Michael S; Tayek, John A (2018) The durability of oral diabetic medications: Time to A1c baseline and a review of common oral medications used by the primary care provider. Endocrinol Diabetes Metab J 2:
Nicholls, Stephen J; Tuzcu, E Murat; Wolski, Kathy et al. (2018) Extent of coronary atherosclerosis and arterial remodelling in women: the NHLBI-sponsored Women's Ischemia Syndrome Evaluation. Cardiovasc Diagn Ther 8:405-413
Wei, Janet; Bakir, May; Darounian, Navid et al. (2018) Myocardial Scar Is Prevalent and Associated With Subclinical Myocardial Dysfunction in Women With Suspected Ischemia But No Obstructive Coronary Artery Disease: From the Women's Ischemia Syndrome Evaluation-Coronary Vascular Dysfunction Study. Circulation 137:874-876
Elboudwarej, Omeed; Wei, Janet; Darouian, Navid et al. (2018) Maladaptive left ventricular remodeling in women: An analysis from the Women's Ischemia Syndrome Evaluation-Coronary Vascular Dysfunction study. Int J Cardiol 268:230-235
Shufelt, Chrisandra; Bairey Merz, C Noel; Pettinger, Mary B et al. (2018) Estrogen-alone therapy and invasive breast cancer incidence by dose, formulation, and route of delivery: findings from the WHI observational study. Menopause 25:985-991
Mehta, Puja K; Hermel, Melody; Nelson, Michael D et al. (2018) Mental stress peripheral vascular reactivity is elevated in women with coronary vascular dysfunction: Results from the NHLBI-sponsored Cardiac Autonomic Nervous System (CANS) study. Int J Cardiol 251:8-13
Kim, Se-Min; Cui, Jinrui; Rhyu, Jane et al. (2018) Association between site-specific bone mineral density and glucose homeostasis and anthropometric traits in healthy men and women. Clin Endocrinol (Oxf) 88:848-855
Nakanishi, Rine; Baskaran, Lohendran; Gransar, Heidi et al. (2017) Relationship of Hypertension to Coronary Atherosclerosis and Cardiac Events in Patients With Coronary Computed Tomographic Angiography. Hypertension 70:293-299

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