Abstract

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 overall objective of this study is to evaluate the safety and efficacy of alternative dosing regimens of Myozyme in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week (qow) after a minimum of 6 months of regular treatment immediately prior to study entry. Eligible patients will be randomized on a 1:1 basis to a """"""""frequent dosing group"""""""" (20 mg/kg every week [qw]) or a """"""""high dose group"""""""" (40 mg/kg every other week [qow]) of Myozyme treatment.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Center for Research Resources (NCRR)
Type
General Clinical Research Centers Program (M01)
Project #
3M01RR020359-05S2
Application #
8167324
Study Section
National Center for Research Resources Initial Review Group (RIRG)
Project Start
2010-01-20
Project End
2010-06-30
Budget Start
2010-01-20
Budget End
2010-06-30
Support Year
5
Fiscal Year
2010
Total Cost
$18,594
Indirect Cost

  Institution

Name
Children's Research Institute
Department
Type
DUNS #
143983562
City
Washington
State
DC
Country
United States
Zip Code
20010

  Publications

Sady, Maegan D; Vaughan, Christopher G; Gioia, Gerard A (2018) Measuring Dynamic Symptom Response in Concussion: Children's Exertional Effects Rating Scale. J Head Trauma Rehabil :
Mullins, Tanya L Kowalczyk; Li, Su X; Bethel, James et al. (2018) Sexually transmitted infections and immune activation among HIV-infected but virally suppressed youth on antiretroviral therapy. J Clin Virol 102:7-11
Kahn, Jessica A; Xu, Jiahong; Kapogiannis, Bill G et al. (2017) Brief Report: Antibody Responses to Quadrivalent HPV Vaccination in HIV-Infected Young Women as Measured by Total IgG and Competitive Luminex Immunoassay. J Acquir Immune Defic Syndr 75:241-245
Smits, Anne; van den Anker, John N; Allegaert, Karel (2017) Clinical pharmacology of analgosedatives in neonates: ways to improve their safe and effective use. J Pharm Pharmacol 69:350-360
Newport, Elissa L; Landau, Barbara; Seydell-Greenwald, Anna et al. (2017) Revisiting Lenneberg's Hypotheses About Early Developmental Plasticity: Language Organization After Left-Hemisphere Perinatal Stroke. Biolinguistics (Nicos) 11:407-422
Gioia, Gerard A (2016) Medical-School Partnership in Guiding Return to School Following Mild Traumatic Brain Injury in Youth. J Child Neurol 31:93-108
Terwilliger, Virginia K; Pratson, Lincoln; Vaughan, Christopher G et al. (2016) Additional Post-Concussion Impact Exposure May Affect Recovery in Adolescent Athletes. J Neurotrauma 33:761-5
Ruan, Alexandra; Tobin, Nicole H; Mulligan, Kathleen et al. (2016) Brief Report: Macrophage Activation in HIV-Infected Adolescent Males Contributes to Differential Bone Loss by Sex: Adolescent Trials Network Study 021. J Acquir Immune Defic Syndr 72:372-5
Orrock, Janet E; Panchapakesan, Karuna; Vezina, Gilbert et al. (2016) Association of brain injury and neonatal cytokine response during therapeutic hypothermia in newborns with hypoxic-ischemic encephalopathy. Pediatr Res 79:742-7
Sepeta, Leigh N; Berl, Madison M; Wilke, Marko et al. (2016) Age-dependent mesial temporal lobe lateralization in language fMRI. Epilepsia 57:122-30

Showing the most recent 10 out of 203 publications