Abstract

Nephropathic cystinosis is an inherited disease with multiple clinical manifestations including Fanconi's syndrome and progressive kidney failure. Within the past decade, cysteamine and phosphocysteamine were developed as orphan drugs. These compounds have now been found to be effective in reducing the rate of progression of cystinosis. However, multiple daily doses of medication and regular monitoring of white blood cell cystine levels are essential to optimal outcomes.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
2M01RR000585-29
Application #
6306855
Study Section
Project Start
1999-12-01
Project End
2000-11-30
Budget Start
Budget End
Support Year
29
Fiscal Year
2000
Total Cost
Indirect Cost

  Institution

Name
Mayo Clinic, Rochester
Department
Type
DUNS #
City
Rochester
State
MN
Country
United States
Zip Code
55905

  Publications

Yu, Alan S L; Shen, Chengli; Landsittel, Douglas P et al. (2018) Baseline total kidney volume and the rate of kidney growth are associated with chronic kidney disease progression in Autosomal Dominant Polycystic Kidney Disease. Kidney Int 93:691-699
Kamimura, Daisuke; Suzuki, Takeki; Wang, Wanmei et al. (2018) Higher plasma leptin levels are associated with reduced left ventricular mass and left ventricular diastolic stiffness in black women: insights from the Genetic Epidemiology Network of Arteriopathy (GENOA) study. Hypertens Res 41:629-638
Nowak, Kristen L; You, Zhiying; Gitomer, Berenice et al. (2018) Overweight and Obesity Are Predictors of Progression in Early Autosomal Dominant Polycystic Kidney Disease. J Am Soc Nephrol 29:571-578
Tirumanisetty, P; Prichard, D; Fletcher, J G et al. (2018) Normal values for assessment of anal sphincter morphology, anorectal motion, and pelvic organ prolapse with MRI in healthy women. Neurogastroenterol Motil 30:e13314
McKenzie, Katelyn A; El Ters, Mirelle; Torres, Vicente E et al. (2018) Relationship between caffeine intake and autosomal dominant polycystic kidney disease progression: a retrospective analysis using the CRISP cohort. BMC Nephrol 19:378
Dad, Taimur; Abebe, Kaleab Z; Bae, K Ty et al. (2018) Longitudinal Assessment of Left Ventricular Mass in Autosomal Dominant Polycystic Kidney Disease. Kidney Int Rep 3:619-624
Brosnahan, Godela M; Abebe, Kaleab Z; Rahbari-Oskoui, Frederic F et al. (2017) Effect of Statin Therapy on the Progression of Autosomal Dominant Polycystic Kidney Disease. A Secondary Analysis of the HALT PKD Trials. Curr Hypertens Rev 13:109-120
Kamimura, Daisuke; Suzuki, Takeki; Furniss, Anna L et al. (2017) Elevated serum osteoprotegerin is associated with increased left ventricular mass index and myocardial stiffness. J Cardiovasc Med (Hagerstown) 18:954-961
Chung, Jin Ook; Koutsari, Christina; Blachnio-Zabielska, Agnieszka U et al. (2017) Intramyocellular Ceramides: Subcellular Concentrations and Fractional De Novo Synthesis in Postabsorptive Humans. Diabetes 66:2082-2091
West, Nancy A; Lirette, Seth T; Cannon, Victoria A et al. (2017) Adiposity, Change in Adiposity, and Cognitive Decline in Mid- and Late Life. J Am Geriatr Soc 65:1282-1288

Showing the most recent 10 out of 1267 publications