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 examines the safety of long-term use of enzyme replacement therapy (replacing the enzyme that, when missing, causes the disease) for MPSII. If proven safe, this drug has the possibility of greatly improving the health and quality of life of all individuals affected with Mucopolysaccharidosis type II (MPS II), a disease for which there is currently no cure. He study design allows patients who successfully completed the phase I/II trial to continue to receive treatment with enzyme replacement therapy, and for patients who had received placebo in the previous trial to cross-over and receive enzyme replacement therapy.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
2M01RR008084-14
Application #
7607743
Study Section
National Center for Research Resources Initial Review Group (RIRG)
Project Start
2007-02-15
Project End
2007-11-30
Budget Start
2007-02-15
Budget End
2007-11-30
Support Year
14
Fiscal Year
2007
Total Cost
$12,814
Indirect Cost

  Institution

Name
Cincinnati Children's Hospital Medical Center
Department
Type
DUNS #
071284913
City
Cincinnati
State
OH
Country
United States
Zip Code
45229

  Publications

Natarajan, Girija; Shankaran, Seetha; Laptook, Abbot R et al. (2018) Association between sedation-analgesia and neurodevelopment outcomes in neonatal hypoxic-ischemic encephalopathy. J Perinatol 38:1060-1067
Askie, Lisa M; Darlow, Brian A; Finer, Neil et al. (2018) Association Between Oxygen Saturation Targeting and Death or Disability in Extremely Preterm Infants in the Neonatal Oxygenation Prospective Meta-analysis Collaboration. JAMA 319:2190-2201
DiFrancesco, Mark W; Shamsuzzaman, Abu; McConnell, Keith B et al. (2018) Age-related changes in baroreflex sensitivity and cardiac autonomic tone in children mirrored by regional brain gray matter volume trajectories. Pediatr Res 83:498-505
Autmizguine, Julie; Tan, Sylvia; Cohen-Wolkowiez, Michael et al. (2018) Antifungal Susceptibility and Clinical Outcome in Neonatal Candidiasis. Pediatr Infect Dis J 37:923-929
Jilling, Tamas; Ambalavanan, Namasivayam; Cotten, C Michael et al. (2018) Surgical necrotizing enterocolitis in extremely premature neonates is associated with genetic variations in an intergenic region of chromosome 8. Pediatr Res 83:943-953
Hahn, Andrew D; Higano, Nara S; Walkup, Laura L et al. (2017) Pulmonary MRI of neonates in the intensive care unit using 3D ultrashort echo time and a small footprint MRI system. J Magn Reson Imaging 45:463-471
Kingery, Kathleen M; Narad, Megan E; Taylor, H Gerry et al. (2017) Do Children Who Sustain Traumatic Brain Injury in Early Childhood Need and Receive Academic Services 7 Years After Injury? J Dev Behav Pediatr 38:728-735
Glauser, Tracy A; Holland, Katherine; O'Brien, Valerie P et al. (2017) Pharmacogenetics of antiepileptic drug efficacy in childhood absence epilepsy. Ann Neurol 81:444-453
Durber, Chelsea M; Yeates, Keith Owen; Taylor, H Gerry et al. (2017) The family environment predicts long-term academic achievement and classroom behavior following traumatic brain injury in early childhood. Neuropsychology 31:499-507
Hung, Anna H; Cassedy, Amy; Schultz, Hanna M et al. (2017) Predictors of Long-Term Victimization After Early Pediatric Traumatic Brain Injury. J Dev Behav Pediatr 38:49-57

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