Funded Grants
Click the drop downs below to learn more about
Rosenau Family Research Foundation’s grant recipients and their work.
The following grants were approved in 2025:
- Enriching CF Newborn Screening Outcomes through a Precision Personalized Medicine Strategy
- Hara Levy, MD, MMSc
- University of Wisconsin – Madison, WI
- Funded $375,000 through 2027
- Molecular characterization and pathogenicity assessment of common variants of uncertain significance in Krabbe Disease
- Chris Lee, PhD
- Biomedical Research Institute of New Jersey
- Funded $375,000 through 2027
- Autologous Transplantation of Genome-Edited Hematopoietic Stem and Progenitors Cells for Krabbe Disease
- Natalia Gomez-Ospina, MD, PhD
- Stanford University
- Funded $375,000 through 2027
- Engineering Adeno-Associated Viral Vectors for Broad Central and Peripheral Nervous System Targeting
- Changfan Lin
- Caltech
- Funded $120,000 through 2027
- Leveraging microglia replacement tools to understand and improve cross-correction in Krabbe disease
- Venkata Sai Chaluvadi
- University of Pennsylvania
- Funded $120,000 through 2027
- Investigating the role of a novel TMEM16A isoform in cystic fibrosis
- Tom Brett, PhD
- Washington University in Saint Louis
- Funded $375,000 through 2028
- GenoKrabbe: A Comprehensive Genotype-Phenotype Database for Krabbe Disease
- Robert Thompson Stone, MD
- University of Rochester
- Funded $155,000 through 2028
The Foundation funded a total of $1,705,137 in grants in 2024.
The following grants were approved in 2024:
- Modulating NF-kB as a therapeutic strategy for Krabbe disease
- Daesung Shin, PhD
- SUNY Buffalo – Amherst, NY
- Funded $250,000 through 2026
- Elucidating the metabolic signature of Krabbe Disease plasma, dried blood spots, and cerebrospinal fluid using untargeted metabolomics and metabolic flux analysis
- Rachel Wurth
- Mayo Clinic – Rochester, MN
- Funded $112,718 through 2026
- Disclosure of Newborn Screening Results for Krabbe Disease in Pennsylvania
- Laura Kirkpatrick, MD
- University of Pittsburgh – Pittsburgh, PA
- Funded $375,000 through 2027
- Evaluation of in utero gene therapy in canine Krabbe disease
- Allison Bradbury, MS, PhD
- Nationwide Children’s Hospital – Columbus, OH
- Funded $375,000
- GenoKrabbe: A Comprehensive Genotype-Phenotype Database for Krabbe Disease
- Paul Orchard, MD
- University of Minnesota Medical School – Minneapolis, MN
- Funded $360,000
The Foundation funded a total of $1,266,475 in grants in 2023.
The following grants were approved in 2023:
- Challenging the Capacity of AAV-GALC Gene Therapy to Cope with Modifiers of Adult Brain Function
- Ernesto Bongarzone, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $255,000 through 2025
- Elucidating the Mechanisms Underlying Macrophage Dysfunction in Krabbe Disease
- Sai (Venkata) Chaluvadi
- University of Pennsylvania – Philadelphia, PA
- Funded $120,000 through 2024
- Immune Responses in Peripheral Nerves After Hematopoietic Stem Cell Transplants for Krabbe
- Anthony Filiano
- Duke University – Durham, NC
- Funded $375,000 through 2025
- A Maladaptive IL-1 f3-Acid Ceramidase-Inflammatory Pathway Involved in Early Pathogenesis of Krabbe Disease
- Maria I. Givogri, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $375,000 through 2025
- Improving Developmental Outcomes in Mouse Models of Krabbe Disease
- Steven LeVine, PhD
- University of Kansas – Lawrence, KS
- Funded $360,000 through 2025
- Parent-Report Disease Burden in Infantile and Late Infantile Krabbe Disease; A Comparison of Quality-of-Life Outcomes in Transplant and Non-or-Late Transplant Patients
- Stacy Pike-Langenfeld
- KrabbeConnect – Rosemount, MN
- Funded $246,690 through 2024
- Effects of Motor Learning on Efficacy and Safety of AAV-Gene Therapy in Krabbe Disease
- Natalia Saldivia Soto, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $120,000 through 2024
- Dysfunction of Brain Synapsis in Krabbe Disease
- Diego Zelada, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $120,000 through 2024
The Foundation funded a total of $383,067 in grants in 2022.
The following grants were approved in 2022:
- Improving the Family Experience through communication and promoting resilience; A pilot project
- Laura A. Adang, MD, PhD
- Children’s Hospital of Philadelphia – Philadelphia, PA
- Funded $81,168 through 2024
- Evaluation of Immunomodulatory Therapy for the Treatment of Krabbe Disease in a Mouse Model
- Stephen Crocker, PhD
- University of Connecticut – Storrs, CT
- Funded $381,589 through 2025
- Psychosine profiling and evaluation of 1-deoxygalactonojirimycin as a therapeutic for Krabbe disease
- Chris Lee, PhD
- Biomedical Research Institute of New Jersey – Cedar Knolls, NJ
- Funded $403,743 through 2024
- Caregiver and Clinician AwaREness for early Cystic Fibrosis diagnosis (C-CARE CF)
- Susanna A. McColley, MD
- Lurie Children’s Hospital – Chicago, IL
- Funded $400,000 through 2023
The Foundation funded a total of $1,472,690 in grants in 2021.
The following grants were approved in 2021:
- Paul M. Fernhoff Memorial Lecture Series
- Dawn Laney, MS, CGC
- Emory University – Atlanta, GA
- Funded $5,000 through 2021
- Support for the Infrastructure of KD & Myelin Research at the University of Buffalo
- Laura M. Feltri, MD
- The Research Foundation for SUNY – Albany, NY
- Funded $381,168 through 2022
- Innovative Model Systems to Study & Boost Innate Immunity in KD
- Laura M. Feltri, MD
- The Research Foundation for SUNY – Albany, NY
- Funded $527,623 through 2023
The Foundation funded a total of $1,988,946 in grants in 2020.
The following grants were approved in 2020:
- Treatment of Krabbe Disease Using Gene Therapy: Rat Toxicology Studies and
Preclinical Drug Production- Maria Luisa Escolar, MD
- Children’s Hospital of Pittsburgh – Pittsburgh, PA
- Funded 943,401 through 2020
- Developing a Target-Assay for Substrate Reduction Agents for Krabbe Disease
- Gustavo Maegawa, MD, PhD
- University of Florida College of Medicine – Gainesville, FL
- Funded $21,000 through 2020
- Brain Connectome as a Novel Biomarker of Krabbe Disease
- Li Ou, PhD
- University of Minnesota – Minneapolis, MN
- Funded $93,548 through 2020
- NDRD Krabbe Research
- Maria Luisa Escolar, MD
- Children’s Hospital of Pittsburgh – Pittsburgh, PA
- Funded $615,899 through 2021
- Vesicular Delivery of GALC for ERT of Krabbe’s disease
- Maria I. Givogri, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $449,999 through 2022
- Generation of a Krabbe Mouse Model to Enable Regulated Expression of Therapeutic
GALC Enzyme- Ernesto Bongarzone, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $225,000 through 2022
The Foundation funded a total of $1,473,072 in grants in 2019.
The following grants were approved in 2019:
- Studies of GALC Correction Stability after AAV Gene Therapy for Krabbe Disease
- Ernesto Bongarzone, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $540,000 through 2021
- Development of High Throughput Screening Assay for Acid Ceramidase and UGT8 Inhibitors
- Michael Gelb, PhD
- University of Washington – Seattle, WA
- Funded $23,397 through 2019
- Optimizing the Treatment of the Mouse Model of Krabbe Disease for Translation into Human Trial
- David A. Wenger, PhD
- Thomas Jefferson University – Philadelphia, PA
- Funded $127,949 through 2019
- The Paul M. Fernhoff Memorial Lecture Series
- Dawn Laney, MS, CGC
- Emory University – Atlanta, GA
- Funded $15,750 through 2019
The Foundation funded a total of $491,807 in grants in 2018.
The following grants were approved in 2018:
- Krabbe Disease Patient Care and Research (NDRD)
- Maria Luisa Escolar, MD
- Children’s Hospital of Pittsburgh – Pittsburgh, PA
- Funded $361,269 through 2019
- Treatment of Krabbe Disease Using Gene Therapy: Continuation
- Maria Luisa Escolar, MD
- Children’s Hospital of Pittsburgh – Pittsburgh, PA
- Funded $174,821 through 2019
- Development of Pharmacological Chaperone Therapy for Krabbe Disease
- Chris Lee, PhD
- Biomedical Research Institute of New Jersey – Cedar Knolls, NJ
- Funded $393,572 through 2020
- Optimizing the Treatment of the Mouse Model of Krabbe Disease for Translation into Human Trial
- David A. Wenger, PhD
- Thomas Jefferson University – Philadelphia, PA
- Funded $125,707 through 2018
The Foundation funded a total of $1,260,317 in grants in 2017.
The following grant was approved in 2017:
- Assessing the Added Value of Whole Genome Sequencing in Cystic Fibrosis Newborn Screening
- Philip Farrell, MD, PhD; Mei Baker, MD
- University of Wisconsin, Madison – Madison, WI
- Funded $717,045 through 2019
The Foundation funded a total of $3,596,417 in grants in 2016.
The following grants were approved in 2016:
- Treatment of Krabbe Disease Using Gene Therapy: Rat Toxicology Studies and Preclinical Drug Production
- Maria Luisa Escolar, MD
- Children’s Hospital of Pittsburgh – Pittsburgh, PA
- Funded $2,199,966 through 2017
- A Pilot Engraftment Study in Immunosuppressed Rats
- Maria Luisa Escolar, MD
- Children’s Hospital of Pittsburgh – Pittsburgh, PA
- Funded $65,472 through 2016
- The Paul M. Fernhoff Memorial Lecture Series
- Dawn Laney, MS, CGC
- Emory University – Atlanta, GA
- Funded $30,000 through 201
The Foundation funded a total of $2,221,332 in grants.
The following grants were approved in 2015:
- Global Correction of Krabbe Disease Via Combined Cell, Gene, and Neuroprotective Therapies
- Ernesto Bongarzone, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $90,000 through 2015
- Generation and Validation of New Humanized Krabbe Disease Mouse and Induced Pluripotent Stem Cell Models
- Ernesto Bongarzone, PhD
- University of Illinois at Chicago – Chicago, IL
- Funded $472,500 through 2017
- Lentiviral-mediated Hematopoietic Stem Cell Gene Therapy for Canine Globoid Cell Leukodystrophy
- Allison Bradbury, PhD
- University of Pennsylvania – Philadelphia, PA
- Funded $226,800 through 2017
- Development Program for the Treatment of Krabbe Disease Using Intravenous Adeno-associated Virus Gene Therapy
- Maria Luisa Escolar, MD
- Children’s Hospital of Pittsburgh – Pittsburgh, PA
- Funded $1,350,705 through 2017
- High Accuracy Galactosylceramidase (GALC) Enzyme Assay for Second Tier Screening of Krabbe Disease
- Michael Gelb, PhD
- University of Washington – Seattle, WA
- Funded $63,000 through 2017
- Comparison of Intrathecal AAV9, AAVrh10 and AAV-Oligo001 in Combination with Bone Marrow Transplant
- Steven Gray, PhD
- University of North Carolina at Chapel Hill – Chapel Hill, NC
- Funded $228,436 through 2016
- 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 – Atlanta, GA
- Funded $210,000 through 2016
- 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, MD
- Mayo Clinic – Rochester, MN
- Funded $180,167 through 2015
The Foundation funded a total of $1,603,663 in grants in 2014.
The following grants were approved in 2014:
- A Prospective Study of Newborn Screening for Cystic Fibrosis Using Novel IRT/Next Generation Sequencing Method
- Mei Baker, MD
- University of Wisconsin – Madison, WI
- Funded $589,732 through 2015
- Global correction of Krabbe disease via combined cell, gene and neuroprotective therapies
- Ernesto Bongarzone, Ph.D.
- University of Illinois at Chicago – Chicago, IL
- Funded $262,500 through 2014
- Roadmap Initiative to Support Gene Therapy for Krabbe Disease
- Maria Luisa Escolar, MD
- University of Pittsburgh Medical Center – Pittsburgh, PA
- Funded $35,179 through 2014
- 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
- Funded $127,097 through 2015
- A Multi-Center Study of a New Method of Sweat Testing: The CF Quantum Sweat Test
- Michael Rock, MD
- University of Wisconsin – Madison, WI
- Funded $394,510 through 2015
- The Paul M. Fernhoff Memorial Lecture Series CDC Newborn Screening Translational Research Initiative
- Robert Vogt, PhD
- Centers for Disease Control & Prevention Foundation – Atlanta, GA
- Funded $24,000 through 2015
The Foundation funded a total of $1,475,013 in grants in 2013.
The following grants were approved in 2013:
- Program for the Study of Neurodevelopment in Rare Disorders
- Maria Luisa Escolar, MD
- University of Pittsburgh Medical Center – Pittsburgh, PA
- Funded $1,706,580 through 2019
- UK Krabbe Disease Scientific Workshop – Travel Grant for International for Krabbe Disease Expert
- David A. Wenger, PhD; Pat Roberts
- Save Babies Through Screening Foundation UK
- Funded $1,503 through 2013
- Identification of Drugs for the Treatment of Krabbe Disease
- Magdalena A. Petryniak, MD
- Oregon Health Sciences University – Portland, OR
- Funded $205,272 through 2015
- Novel Approaches to the Treatment of Krabbe Disease
- Mark Noble, PhD
- University of Rochester Medical Center – Rochester NY
- Funded $150,000 through 2013
The Foundation funded a total of $1,332,994 in grants in 2012.
The following grants were approved in 2012:
- Intracerebroventricular and Intravenious Injections of AAVrh10-CGALC into the Dog Model of Krabbe Disease
- David A. Wenger, PhD
- Thomas Jefferson University – Philadelphia, PA
- Funded $674,988 through 2014
- Improving IRT/DNA Newborn Screening for Cystic Fibrosis to Reduce False Positives by a New Molecular Strategy
- Mei Baker, MD
- University of Wisconsin – Madison, WI
- Funded $415,273 through 2013
The Foundation funded a total of $1,576,253 in grants in 2011.
The following grants were approved in 2011:
- MRI for Neurodevelopment For Rare Diseases Program
- Maria Luisa Escolar, MD
- University of Pittsburgh Medical Center – Pittsburgh, PA
- Funded $1,250,000 through 2013
- Krabbe Translational Research Network Collaborative Lab Grant
- Maria Luisa Escolar, MD
- University of Pittsburgh Medical Center – Pittsburgh, PA
- Funded $772,225 through 2013
- Quality Improvement in Cystic Fibrosis Newborn Screening: Improving Follow-up and Outcomes with Tools for “Facilitative Interactive Communication
- Michael Farrell, MD
- Medical College of Wisconsin – Milwaukee, WI
- Funded $222,196 through 2012
- Newborn Screening Education Video
- Jill Levy-Fisch
- Save Babies Through Screening Foundation Inc.
- Funded $200,000 through 2011
- Quality Improvement in Cystic Fibrosis Newborn Screening
- Michael Rock, MD
- University of Wisconsin – Madison, WI
- Funded $153,944 through 2011
The Foundation funded a total of $1,320,000 in grants in 2010.
The following grants were approved in 2010:
- Krabbe Transitional Research Network (KTRN) Meeting
- Maria Luisa Escolar, MD
- University of North Carolina at Chapel Hill – Chapel Hill, NC
- Funded $410,577 through 2017
- Development of Efficient and Effective Screening Strategies for Lysosomal Storage Disorders, Friedreich Ataxia, Wilson Disease, & X-Adrenoleukodystrophy
- Dieter Matern, MD, PhD
- Mayo Clinic – Rochester, MN
- Funded $289,000 through 2010
The Foundation funded a total of $500,000 in grants in 2009.
The following grants were approved in 2009:
- Cystic Fibrosis Care and Therapeutics Development in Minnesota
- Cystic Fibrosis Foundation – Bethesda, MD
- Funded $500,000 through 2010
- Infantile Krabbe Disease Research Program
- Joanne Kurtzberg, MD
- Duke University Medical Center – Durham, NC
- Funded $750,00 through 2011
- A Virtual Medical Home and Clinic for Children with Krabbe Disease
- Maria Luisa Escolar, MD
- University of North Carolina at Chapel Hill – Chapel Hill, NC
- Funded $500,000 through 2010