This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The objective of the Myozyme Temporary Access Program is to provide adult patients with Pompe disease in the US access to Myozyme produced from a scaled up manufacturing process for a limited time until production at this scale is approved for commercial use by the Food and Drug Administration (FDA). Myozyme has been used since early 2003 in clinical trials to treat patients with Pompe disease. Market approval in the US for the use of Myozyme to treat Pompe disease was granted on 28 April 2006. Genzyme has scaled up and modified the existing manufacturing process for production of Myozyme at a larger scale, which is currently approved for commercial use outside of the US, in order to provide a continued supply to patients. This scale of material is not yet pproved for commercial use in the US;however it is used in ongoing clinical trials. Myozyme, produced from the scaled-up manufacturing process will be administered for a limited time under this access program until the manufacturing scale is approved by the FDA.

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
5M01RR000827-35
Application #
8166856
Study Section
National Center for Research Resources Initial Review Group (RIRG)
Project Start
2009-12-01
Project End
2010-06-30
Budget Start
2009-12-01
Budget End
2010-06-30
Support Year
35
Fiscal Year
2010
Total Cost
$11,045
Indirect Cost
Name
University of California San Diego
Department
Type
Schools of Medicine
DUNS #
804355790
City
La Jolla
State
CA
Country
United States
Zip Code
92093
Grams, Morgan E; Sang, Yingying; Ballew, Shoshana H et al. (2018) Predicting timing of clinical outcomes in patients with chronic kidney disease and severely decreased glomerular filtration rate. Kidney Int 93:1442-1451
Lavigne, Katie M; Woodward, Todd S (2018) Hallucination- and speech-specific hypercoupling in frontotemporal auditory and language networks in schizophrenia using combined task-based fMRI data: An fBIRN study. Hum Brain Mapp 39:1582-1595
Milot, Marie-Hélène; Marchal-Crespo, Laura; Beaulieu, Louis-David et al. (2018) Neural circuits activated by error amplification and haptic guidance training techniques during performance of a timing-based motor task by healthy individuals. Exp Brain Res 236:3085-3099
Hsu, Simon; Rifkin, Dena E; Criqui, Michael H et al. (2018) Relationship of femoral artery ultrasound measures of atherosclerosis with chronic kidney disease. J Vasc Surg 67:1855-1863.e1
Inker, Lesley A; Grams, Morgan E; Levey, Andrew S et al. (2018) Relationship of Estimated GFR and Albuminuria to Concurrent Laboratory Abnormalities: An Individual Participant Data Meta-analysis in a Global Consortium. Am J Kidney Dis :
Egnot, Natalie Suder; Barinas-Mitchell, Emma; Criqui, Michael H et al. (2018) An exploratory factor analysis of inflammatory and coagulation markers associated with femoral artery atherosclerosis in the San Diego Population Study. Thromb Res 164:9-14
Juraschek, Stephen P; Miller 3rd, Edgar R; Appel, Lawrence J (2018) Orthostatic Hypotension and Symptoms in the AASK Trial. Am J Hypertens 31:665-671
Chen, Teresa K; Appel, Lawrence J; Grams, Morgan E et al. (2017) APOL1 Risk Variants and Cardiovascular Disease: Results From the AASK (African American Study of Kidney Disease and Hypertension). Arterioscler Thromb Vasc Biol 37:1765-1769
Juraschek, Stephen P; Appel, Lawrence J; Miller 3rd, Edgar R (2017) Metoprolol Increases Uric Acid and Risk of Gout in African Americans With Chronic Kidney Disease Attributed to Hypertension. Am J Hypertens 30:871-875
Chen, Teresa K; Tin, Adrienne; Peralta, Carmen A et al. (2017) APOL1 Risk Variants, Incident Proteinuria, and Subsequent eGFR Decline in Blacks with Hypertension-Attributed CKD. Clin J Am Soc Nephrol 12:1771-1777

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