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• Research project

  A Double-Blind, Randomized, Placebo-Controlled, 28-Day S

  Milla, C.

  Inspire Pharmaceuticals, Inc.

  1/15/06 → 1/14/07

  Project: Research project

  • denufosol tetrasodium
  • Inhalation
  • Lung Diseases
  • Placebos
  • Pediatrics

• An open-label, multinational, multicenter, intravenous infusion study of the efficacy, safety, pharmacokinetics and pharmacodynamics of avalglucosidase alfa in treatment-naive

  Berry, S. A.

  SANOFI-AVENTIS US

  3/31/21 → 12/31/23

  Project: Research project

  • Efficacy
  • Safety

• A Phase 3B, Multicenter, Randomized, Double-Masked, Para

  Bothun, E. D.

  ALCON LABORATORIES, INC.

  7/1/10 → 12/31/11

  Project: Research project

  • emulsion
  • acetate

• United States Renal Data System Coordinating Center Subc

  Chavers, B. M.

  HENNEPIN HEALTHCARE RESEARCH INSTITUTE

  2/8/07 → 2/7/14

  Project: Research project

• Center for Modeling Tumor Cell Migration Mechanics

  Odde, D. J., Alford, P. W., Barocas, V. H., Largaespada, D. A., Olin, M. R., Othmer, H. G., Provenzano, P., Schwertfeger, K., Tolar, J., Tranquillo, R. T. & Wood, D. K.

  NIH NATIONAL CANCER INSTITUTE (NCI)

  8/17/16 → 7/31/21

  Project: Research project

  • Tumors
  • Cells
  • Mechanics

• High through-put combinatorial drug and target discovery

  Largaespada, D. A.

  Boston Children's Hospital

  12/1/17 → 11/30/20

  Project: Research project

  • Neurofibrosarcoma
  • Drug Discovery

• Pre-Faculty Research Training Grant in Pediatric Endocri

  Moran, A.

  NIH Natl Inst of Diabetes/Dig/Kidney Dis

  7/1/04 → 7/1/14

  Project: Research project

  • Endocrinology
  • Pediatrics

• A Phase 2 Study of the JAK1/JA2 Inhibitor Ruxolitinib

  Gordon, P. M.

  Children's Hospital of Philadelphia

  7/1/16 → 1/31/22

  Project: Research project

  • N-carboxy-3-phenylpropyl-alanyl-aminoisobutyric acid-tyrosyl-aminobenzoate
  • INCB018424
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Drug Therapy
  • Child

• Preclinical Evaluation of a Novel rLCMV Single-Cycled Ve

  Schleiss, M. R. & Lokensgard, J. R.

  NIH Nat'l Inst of Allergy & Infect Dis

  8/27/15 → 2/28/18

  Project: Research project

  • Vaccines

• Subunit gB Vaccination and Protection Against Congenital

  Schleiss, M. R.

  GlaxoSmithKline

  12/17/07 → 6/30/10

  Project: Research project

  • Roseolovirus
  • Cytomegalovirus Infections
  • Cytomegalovirus
  • Vaccination

• Microstructural and functional MRI signatures in patient

  Nestrasil, I. & Svatkova, A.

  University of Pennsylvania

  1/1/16 → 12/31/16

  Project: Research project

  • Mucopolysaccharidosis I
  • Magnetic Resonance Imaging

• Autism Spectrum Disorders and Intellectual Disability Pr

  Hewitt, A. S. & Esler, A. N.

  Centers for Disease Control and Prevention

  1/1/15 → 12/31/19

  Project: Research project

  • Autism Spectrum Disorder
  • Intellectual Disability

• University of Minnesota Zebrafish Core Facility

  Schimmenti, L. A.

  MN MED FDN

  7/1/09 → 6/30/11

  Project: Research project

• An Observational Study of the Clinical Characteristics a

  Whitley, C. B.

  PREMIER RESEARCH INTERNATIONAL LLC

  4/1/11 → 4/1/13

  Project: Research project

  • Lysosomal acid lipase deficiency
  • Cholesterol Ester Storage Disease
  • Disease Progression
  • Observational Studies
  • Phenotype

• Observing the disease course in the natural history of S

  Shapiro, E. G.

  SHIRE HUMAN GENETIC THERAPIES, INC.

  12/1/13 → 6/30/17

  Project: Research project

  • Mucopolysaccharidosis III

• Vaccines for Prevention of Experimental Congenital CMV

  Schleiss, M. R.

  Vical Incorporated

  2/1/11 → 3/17/13

  Project: Research project

  • Vaccines

• Per Case Reimbursement: COG NCORP Research Base

  Neglia, J. P.

  Children's Hospital of Philadelphia

  8/1/15 → 7/31/19

  Project: Research project

• A Multicenter, Observational Registry of Pediatric Femal

  Miller, B. S.

  ENDO PHARMACEUTICALS INC

  8/1/13 → 7/31/18

  Project: Research project

  • acetates

• Hyundai Hope on Wheels Impact Grant

  Gershan, L. A.

  Hope on Wheels Hyundai Dealers

  12/1/18 → 11/30/19

  Project: Research project

• A Phase 2 Study of Inotuzumab Ozogamicin in Children and Young Adults with Relapsed or Refractory CD22+ B-Acute Lymphoblastic Leukemia (B-ALL)

  Gordon, P. M.

  Children's Hospital of Philadelphia

  2/27/19 → 2/28/22

  Project: Research project

  • Inotuzumab Ozogamicin
  • Precursor Cell Lymphoblastic Leukemia-Lymphoma
  • Young Adult
  • Child

• Moringa oleifera: A common tree to prevent childhood iro

  Cusick, S.

  INTERNATIONAL PEDIATRIC RESEARCH FDN

  11/1/14 → 5/31/16

  Project: Research project

  • Moringa oleifera
  • Iron
  • Brain

• A randomized, phase II placebo-controlled study of the u

  Moertel, C. L. & Mulrooney, D.

  University of South Florida

  6/1/11 → 5/31/12

  Project: Research project

  • Modafinil
  • Methylphenidate
  • Sleepiness
  • Placebos
  • Child

• Child and Teen Checkups: Adolescent Encounter

  Sylvester, M.

  MN DEPARTMENT OF HEALTH

  4/6/10 → 6/30/10

  Project: Research project

  • medicine
  • staff
  • adolescent
  • health

• Coordinating Center for Research to Promote the Health of Children with Birth Defects and People with Developmental and Other Disabilities (Year 3)

  Schleiss, M. R. & Osterholm, E. A.

  University of South Carolina

  9/30/19 → 9/29/21

  Project: Research project

  • Disabled Persons

• Bcl-2 protein Noxa in human T cell metabolism and differentiation

  Kelekar, A., Moriarity, B. S., Pennell, C. A. & Webber, B. R.

  NIH Nat'l Inst of Allergy & Infect Dis

  6/18/20 → 5/31/22

  Project: Research project

  • Noxae
  • Cell Differentiation
  • T-Lymphocytes
  • Proteins

• Exosomal mRNA Signatures to Inform Prognosis of Patients with Osteosarcoma

  Modiano, J., Kim, J. H., Rendahl, A., Spector, L. G. & Weigel, B. J.

  VENN FOUNDATION

  6/1/20 → 6/30/23

  Project: Research project

  • Osteosarcoma
  • Messenger RNA

• Gene Editing of Type VII Collagen Mutations: Moving Towards Clinical Translation

  Tolar, J.

  VARIOUS SPONSORS - NON-FEDERAL

  9/30/13 → 9/30/23

  Project: Research project

  • Collagen Type VII
  • Gene Editing
  • Mutation

• A Switch-Over, Open-Label Study of the Safety, Pharmacok

  Berry, S. A.

  Hyperion Therapeutics

  4/7/11 → 8/1/13

  Project: Research project

  • glycerol phenylbutyrate
  • Inborn Urea Cycle Disorder
  • Pharmacokinetics
  • Pediatrics
  • Safety

• A Phase I, Open-Label, Dose-Escalation Study to Evaluate

  Whitley, C. B.

  BIOMARIN PHARMACEUTICAL, INC.

  12/18/07 → 12/31/09

  Project: Research project

  • Phenylketonurias
  • Pharmacokinetics
  • Safety

• Region 4 Long Term Follow Up and Clinical Outcomes: Inbo

  Berry, S. A. & Mertens, A.

  Michigan Public Health Institute

  6/1/07 → 6/30/11

  Project: Research project

  • Inborn Errors Metabolism

• A Phase lll, Randomized, Controlled Investiagtion of Six

  Han, J. Y.

  University of Alabama at Birmingham

  8/15/08 → 7/31/13

  Project: Research project

  • Valganciclovir
  • Cytomegalovirus Infections
  • Therapeutics

• Building A Dental Home Network for Children with Special

  Scal, P. B.

  Robert Wood Johnson Foundation

  9/1/16 → 8/31/19

  Project: Research project

  • health care

• intrathecal recombinant human N-acetylgalactosamine 4-su

  Whitley, C. B.

  BIOMARIN PHARMACEUTICAL, INC.

  11/6/09 → 12/31/11

  Project: Research project

  • N-Acetylgalactosamine-4-Sulfatase
  • Mucopolysaccharidosis VI
  • Spinal Cord Compression

• Development of myeloid-derived suppressor cells as a the

  Blazar, B. R.

  Boehringer-Ingelheim Pharmaceuticals

  5/27/11 → 4/5/13

  Project: Research project

  • Myeloid-Derived Suppressor Cells

• A Phase 2, Open-label, Multinational Clinical Study to E

  Whitley, C. B.

  BIOMARIN PHARMACEUTICAL, INC.

  3/15/12 → 6/4/12

  Project: Research project

  • human GALNS protein
  • Mucopolysaccharidosis IV
  • Mucopolysaccharidoses
  • Clinical Studies
  • Pediatrics

• Preclinical Study of Therapeutic Notch Inhibition in Chr

  Blazar, B. R.

  University of Michigan

  10/1/14 → 9/30/17

  Project: Research project

  • Graft vs Host Disease
  • Therapeutics

• Measurement of infant feet using Artec Spider 3D Scanner

  Fischer, G.

  Koronis Biomedical Technologies Corp.

  1/1/17 → 6/30/17

  Project: Research project

• COMMITTEE LEADERSHIP: NIH National Clinical Trials Netwo

  Poynter, J.

  Children's Hospital of Philadelphia

  3/1/16 → 2/28/17

  Project: Research project

  • Clinical Trials

• Multi-family Group Psychoeducation for Young Adults with

  Hudock, R. & Esler, A. N.

  University of Wisconsin-Madison

  8/1/16 → 7/31/18

  Project: Research project

  • Young Adult

• Central Corneal Thickness in a Normal Pediatric Populati

  Bothun, E. D., Anderson, J. S., Christiansen, S. & Summers, C. G.

  Jaeb Center for Health Research

  6/1/08 → 12/31/09

  Project: Research project

  • Pediatrics
  • Prospective Studies
  • Child
  • Population

• DSMB Consulting Agreement

  Rheault, M. N.

  CTI CLINICAL TRIAL SERVICES, INC.

  9/14/18 → 12/31/22

  Project: Research project

• Islets, Bone & Sex Hormones: Advancing Human Islet Transplant for Type 1 Diabetes

  Bellin, M. D.

  UNIVERSITY OF ILLINOIS CHICAGO

  1/1/17 → 12/31/18

  Project: Research project

  • Gonadal Steroid Hormones
  • Type 1 Diabetes Mellitus
  • Transplants
  • Bone and Bones

• Inborn Errors of Metabolism Research Fund

  Berry, S. A.

  CHILDREN'S RESEARCH INSTITUTE

  8/1/18 → 7/31/19

  Project: Research project

• Amgen 20140444: A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-Group Study toEvaluate the Safety and Efficacy of Denosumab in Pediatric Subjects With Glucocorticoid-inducedOsteoporosis

  Miller, B. S.

  Amgen Inc.

  8/1/18 → 7/31/22

  Project: Research project

  • Denosumab
  • Glucocorticoids
  • Osteoporosis
  • Placebos
  • Pediatrics

• Molecular Pathogenesis of MLL-Fusion Gene Leukemia

  Kumar, A.

  NIH NATIONAL CANCER INSTITUTE (NCI)

  7/25/07 → 6/30/09

  Project: Research project

  • gene fusion
  • leukemia
  • pathogenesis

• A Phase 3, Randomized, Double-Blind, Placebo-Controlled,

  Whitley, C. B.

  GENZYME CORPORATION

  7/1/10 → 12/31/14

  Project: Research project

  • eliglustat
  • Gaucher Disease
  • Placebos
  • Safety

• Placebo Controlled Trial Evaluating Gabapentin for the T

  Jarnes, J. R. & Hazaert, J.

  GENZYME CORPORATION

  2/23/12 → 12/31/14

  Project: Research project

  • Fabry Disease
  • Gabapentin
  • Neuralgia
  • Placebos
  • Therapeutics

• Proposal for an International Epidemiological Registry for Neonatal Purpura Fulminans

  Boucher, A.

  3/10/20 → …

  Project: Research project

• NCI-COG Pediatric MATCH (Molecular Analysis for Therapy Choice - Screening Protocol) (APEC1621SC)

  Greengard, E. G.

  Children's Hospital of Philadelphia

  7/1/17 → 6/30/21

  Project: Research project

  • Pediatrics
  • Therapeutics

• Liver Tissue Cell Distribution System

  Gourley, G. R. & Sharp, H.

  NIH Natl Inst of Diabetes/Dig/Kidney Dis

  6/29/07 → 6/28/12

  Project: Research project

  • liver
  • tissues
  • cells