Funded Grants

2016

The Foundation funded $3,631,146 in grants for the following:

  • Global Correction of Krabbe Disease Via Combined Cell, Gene and Neuroprotective Therapies
    Ernesto Bongarzone, Ph.D. -- University of Illinois
  • Generation and Validation of New Humanized Krabbe Disease Mouse and Induced Pluripotent Stem Cell Models
    Ernesto Bongarzone, Ph.D. -- University of Illinois
  • Lentiviral-mediated Hematopoietic Stem Cell Gene Therapy for Canine Globoid Cell Leukodystrophy
    Allison Bradbury, Ph.D. -- University of Pennsylvania
  • Krabbe Disease Patient Care and Research (NDRD)
    Maria Escolar, M.D. -- Children’s Hospital of Pittsburgh
  • Development Program for the Treatment of Krabbe Disease Using Intravenous Adeno-associated Virus Gene Therapy
    Maria Escolar, M.D. -- Children’s Hospital of Pittsburgh
  • Treatment of Krabbe Disease Using Gene Therapy: Rat Toxicology Studies and Preclinical Drug Production
    Maria Escolar, M.D. -- Children’s Hospital of Pittsburgh
  • A Pilot Engraftment Study in Immunosuppressed Rats
    Maria Escolar, M.D. -- Children’s Hospital of Pittsburgh
  • High Accuracy Galactosylceramidase (GALC) Enzyme Assay for Second Tier Screening of Krabbe Disease
    Michael Gelb, Ph.D. -- University of Washington
  • Comparison of Intrathecal AAV9, AAVrh10 and AAV-Oligo001 in Combination with Bone Marrow Transplant
    Steven Gray, Ph.D. -- University of North Carolina at Chapel Hill
  • GeneThink: A Revolutionary Educational Website/Application Providing Increased Access to Accurate and updated Natural History, Diagnosis, and Treatment Information About Genetic Disorders
    Dawn Laney, MS, CGC -- Emory University
  • The Paul M. Fernhoff Memorial Lecture Series
    Dawn Laney, MS, CGC -- Emory University

2015

The Foundation funded $2,221,332.00 in grants for the following:

  • A Prospective Study of Newborn Screening for Cystic Fibrosis Using A Novel IRT/Next Generation Sequencing Method
    Mei Baker, M.D. -- University of Wisconsin
  • Global Correction of Krabbe Disease Via Combined Cell, Gene, and Neuroprotective Therapies
    Ernesto Bongarzone, Ph.D. -- University of Illinois
  • Generation and Validation of New Humanized Krabbe Disease Mouse and Induced Pluripotent Stem Cell Models
    Ernesto Bongarzone, Ph.D. -- University of Illinois
  • Lentiviral-mediated Hematopoietic Stem Cell Gene Therapy for Canine Globoid Cell Leukodystrophy
    Allison Bradbury, Ph.D. -- University of Pennsylvania
  • Krabbe Disease Patient Care and Research (NDRD)
    Maria Escolar, M.D. -- Children’s Hospital of Pittsburgh
  • Development Program for the Treatment of Krabbe Disease Using Intravenous Adeno-associated Virus Gene Therapy
    Maria Escolar, M.D. -- Children’s Hospital of Pittsburgh
  • KTRN Annual Research Meeting
    Maria Escolar, M.D. -- Children’s Hospital of Pittsburgh
  • High Accuracy Galactosylceramidase (GALC) Enzyme Assay for Second Tier Screening of Krabbe Disease
    Michael Gelb, Ph.D. -- University of Washington
  • Comparison of Intrathecal AAV9, AAVrh10 and AAV-Oligo001 in Combination with Bone Marrow Transplant
    Steven Gray, Ph.D. -- University of North Carolina at Chapel Hill
  • Low IRT Levels and Characteristics of Persons with False Negative Newborn Screening Tests for Cystic Fibrosis in the United States
    Martin Kharazzi, Ph.D. -- Sequoia Foundation
  • GeneThink: A Revolutionary Educational Website/Application Providing Increased Access to Accurate and updated Natural History, Diagnosis, and Treatment Information About Genetic Disorders
    Dawn Laney, MS, CGC -- Emory University
  • Completion of a Study to Develop Efficient and Effective Screening Strategies for Lysosomal Storage Disorders, Friedeich Ataxia, Wilson Disease and X-linked Adrenoleukodystrophy
    Dietrich Matern, M.D. -- Mayo Clinic
  • Identification of Drugs for the Treatment of Krabbe's Disease
    Magdalena Petryniak, M.D. -- Oregon Health and Science University Foundation
  • A Multi-Center Study of a New Method of Sweat Testing: The CF Quantum Sweat Test
    Michael Rock, M.D. -- University of Wisconsin
  • The Paul M. Fernhoff Memorial Lecture Series
    Robert Vogt, Ph.D. --CDC Foundation

 

2014

The Foundation funded $1,602,663 in grants for the following:

  • A Prospective Study of Newborn Screening for Cystic Fibrosis Using Novel IRT/Next Generation Sequencing Method
    Mei Baker, M.D. – University of Wisconsin, Madison, WI
  • Global correction of Krabbe’s Disease via combined cell, gene and neuroprotective therapies
    Ernesto Bongarzone, Ph.D. – University of Illinois at Chicago, Chicago, IL
  • Krabbe Translational Research Network (KTRN) Annual Meeting
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Roadmap Initiative to Support Gene Therapy for Krabbe’s Disease
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Program for the Study of Neurodevelopment in Rare Disorders
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Low IRT Levels and Characteristics of Persons with False Negative Newborn Screening Tests for Cystic Fibrosis in the United States
    Martin Kharrazi, Ph.D. – CA Dept. of Public Health/Genetic Disease Screening Program, Richmond, CA
  • Identification of Drugs for the Treatment of Krabbe Disease
    Magdalena A. Petryniak, M.D. – Oregon Health Sciences University, Portland OR
  • A Multi-Center Study of a New Method of Sweat Testing: The CF Quantum Sweat Test
    Michael Rock, M.D. – University of Wisconsin, Madison, WI
  • Intracerebroventricular and Intravenous Injections of AAVrh10-CGALC into the Dog Model of Krabbe Disease
    David Wenger, Ph.D. – Thomas Jefferson University, Philadelphia, PA
  • The Paul M. Fernhoff Memorial Lecture Series CDC Newborn Screening Translational Research Initiative
    Centers for Disease Control & Prevention Foundation, Atlanta, GA

2013

The Foundation funded $1,475,013 in grants for the following:

  • Krabbe Translational Research Network (KTRN) Annual Meeting
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Krabbe Translational Research Network (KTRN) Collaborative Lab
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Program for the Study of Neurodevelopment in Rare Disorders
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • MRI for Program for the Study of Neurodevelopment in Rare Disorders
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh PA
  • Improving IRT/DNA Newborn Screening for Cystic Fibrosis to Reduce False Positives by a New Molecular Strategy 
    Mei Baker, M.D. – University of Wisconsin, Madison WI
  • UK Krabbe Disease Scientific Workshop - Travel Grant for International for Krabbe Disease Expert 
    Pat Roberts – Save Babies Through Screening Foundation UK 
  • Identification of Drugs for the Treatment of Krabbe Disease
    Magdalena A. Petryniak, M.D. – Oregon Health Sciences University, Portland OR
  • Novel Approaches to the Treatment of Krabbe Disease
    Mark Noble, Ph.D. – University of Rochester Medical Center, Rochester NY
  • Intracerebroventricular and Intravenous Injections of AAVrh10-CGALC into the Dog Model of Krabbe Disease
    David Wenger Ph.D. – Thomas Jefferson University, Philadelphia, PA

2012

The Foundation funded $1,332,994 in grants for the following:

  • MRI for Neurodevelopment For Rare Diseases Program
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Krabbe Translational Research Network Collaborative Lab Grant
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Krabbe Transitional Research Network (KTRN) Meeting
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Intracerebroventricular and Intravenious Injections of AAVrh10-CGALC into the Dog Model of Krabbe Disease
    David Wenger, Ph.D. - Thomas Jefferson University, Philadelphia, PA
  • Quality Improvement in Cystic Fibrosis Newborn Screening: Improving Follow-up and Outcomes with Tools for Facilitative Interactive Communication
    Michael Farrell, M.D. - Medical College of Wisconsin, Milwaukee, Wisconsin
  • Improving IRT/DNA Newborn Screening for Cystic Fibrosis to Reduce False Positives by a New Molecular Strategy
    Mei Baker, M.D. - University of Wisconsin, Madison, Wisconsin

2011

The Foundation funded $1,576,253 in grants for the following:

  • MRI for Neurodevelopment For Rare Diseases Program
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA 
  • Krabbe Translational Research Network Collaborative Lab Grant
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Krabbe Transitional Research Network (KTRN) Meeting
    Maria Luisa Escolar, M.D. – University of Pittsburgh Medical Center, Pittsburgh, PA
  • Quality Improvement in Cystic Fibrosis Newborn Screening: Improving Follow-up and Outcomes with Tools for “Facilitative Interactive Communication
    Michael Farrell, M.D. - Medical College of Wisconsin, Milwaukee, Wisconsin
  • Newborn Screening Education Video
    Jill Levy-Fisch – Save Babies Through Screening Foundation Inc.
  • Infantile Krabbe Disease Research Program
    Joanne Kurtzberg, M.D. - Duke University Medical Center, Durham, North Carolina 
  • Quality Improvement in Cystic Fibrosis Newborn Screening
    Michael Rock, M.D. - University of Wisconsin, Madison, Wisconsin

2010

The Foundation funded $1,289,000 in grants for the following:

  • A Virtual Medical Home and Clinic for Children with Krabbe Disease
    Maria Luisa Escolar, M.D. - University of North Carolina - Chapel Hill, North Carolina
  • Krabbe Transitional Research Network (KTRN) Meeting
    Maria Luisa Escolar, M.D. - University of North Carolina - Chapel, Hill, North Carolina
  • Infantile Krabbe Disease Research Program
    Joanne Kurtzberg M.D. - Duke University Medical Center, Durham, North Carolina
  • Development of Efficient and Effective Screening Strategies for Lysosomal Storage Disorders, Friedreich Ataxia, Wilson Disease, & X-Adrenoleukodystrophy
    Dieter Matern, M.D., Ph.D. - Mayo Clinic, Rochester, Minnesota
  • "Molecular Defects of CFTR caused by the DF508 Mutation"
    The Cystic Fibrosis Foundation

2009

The Foundation funded $500,000 in grants for the following:

  • Cystic Fibrosis Care and Therapeutics Development in Minnesota
    Cystic Fibrosis Foundation, Bethesda, Maryland
  • Infantile Krabbe Disease Research Program
    Joanne Kurtzberg M.D. - Duke University Medical Center, Durham, North Carolina