Awarded Grants
Awarded Grants
Mechanistic and Therapeutic Studies of Fibrous Dysplasia Craniofacial Defects
Yingzi Yang
Harvard Medical School
$54,663
Awardee: Yingzi Yang
Institution: Harvard Medical School
Award Amount: $54,663
Assessment of Disease Activity in FOP Patients using Electrical Impedance Myography
Jaymin Upadhyay
Boston Children's Hospital, Harvard Medical School
$40,208
Awardee: Jaymin Upadhyay
Institution: Boston Children's Hospital, Harvard Medical School
Award Amount: $40,208
Tissue-specific and temporal reinstatement of Tcf4 function to treat Pitt-Hopkins Syndrome
Andrew Kennedy
Bates College
$51,242
Awardee: Andrew Kennedy
Institution: Bates College
Award Amount: $51,242
Funding Period: February 1, 2019 - January 31, 2020
Pilot study to develop a small molecule activator of TCF4 as a treatment for Pitt-Hopkins syndrome
Ben Philpot
University of North Carolina, Chapel Hill
$51,242
Awardee: Ben Philpot
Institution: University of North Carolina, Chapel Hill
Award Amount: $51,242
Funding Period: February 1, 2019 - January 31, 2020
Gene Replacement Therapy for APBD
Berge Minassian
UT Southwestern Medical Center
$53,455
Awardee: Berge Minassian
Institution: UT Southwestern Medical Center
Award Amount: $53,455
Funding Period: February 1, 2019 - January 31, 2020
Targeting phagosome maturation to restore dysfunctional retinal pigmented epithelium in CHM
Kathleen Boesze-Battaglia
University of Pennsylvania
$101,740
Awardee: Kathleen Boesze-Battaglia
Institution: University of Pennsylvania
Award Amount: $101,740
Funding Period: February 1, 2019 - January 31, 2020
AAV gene therapy on cultured human iPSC-derived retina with loss of CRB1 and CRB2
Jan Winjholds
Leiden University Medical Center (LUMC)
$50,312
Awardee: Jan Winjholds
Institution: Leiden University Medical Center (LUMC)
Award Amount: $50,312
Funding Period: February 1, 2019 - January 31, 2020
CRB1-null human retinal organoids for disease modeling and gene therapy testing
Wei Liu
Albert Einstein College of Medicine
$50,312
Awardee: Wei Liu
Institution: Albert Einstein College of Medicine
Award Amount: $50,312
Funding Period: February 1, 2019 - January 31, 2020
Role of the GM-CSF Pathway in Heterotypic Ossification Associated with FOP
Eileen Shore
University of Pennsylvania
$37,633
Awardee: Eileen Shore
Institution: University of Pennsylvania
Award Amount: $37,633
Funding Period: February 1, 2019 - January 31, 2020
Advancing A Novel Therapeutic Lead for RASopathies
Bruce Gelb
Icahn School of Medicine at Mount Sinai
$74,830
Awardee: Bruce Gelb
Institution: Icahn School of Medicine at Mount Sinai
Award Amount: $74,830
Funding Period: February 1, 2019 - January 31, 2020
Repurposing FDA-approved drugs for Niemann Pick C Disease
Yamuna Krishnan
University of Chicago
$62,340
Awardee: Yamuna Krishnan
Institution: University of Chicago
Award Amount: $62,340
Muscle Directed Gene Therapy Application for Maple Syrup Urine Disease (MSUD)
Jenny Greig
Gene Therapy Program, University of Pennsylvania
$112,893
Awardee: Jenny Greig
Institution: Gene Therapy Program, University of Pennsylvania
Award Amount: $112,893
Funding Period: February 1, 2019 - January 31, 2020
TFEB and Lysosomal exocytosis to prevent lung destruction in LAM
Elizabeth Henske
Brigham and women's Hospital
$50,242
Awardee: Elizabeth Henske
Institution: Brigham and women's Hospital
Award Amount: $50,242
Funding Period: February 1, 2019 - January 31, 2020
Analysis of crosstalk between regulatory T and bone cells in Gorham-Stout disease
Andrea Del Fattore
Bambino Gesù Children’s Hospital, IRCCS
$50,535
Awardee: Andrea Del Fattore
Institution: Bambino Gesù Children’s Hospital, IRCCS
Award Amount: $50,535
Funding Period: February 1, 2019 - January 31, 2020
Role of NRAS mutations in the pathology of generalized Lymphangiomatosis anaomaly (GLA)
Shoshana Greenberger
Sheba Medical Center and Tel-Aviv University
$50,535
Awardee: Shoshana Greenberger
Institution: Sheba Medical Center and Tel-Aviv University
Award Amount: $50,535
Funding Period: February 1, 2019 - January 31, 2020
Could exon skipping strategies be used as a treatment for recessive Ullrich Congenital Muscular Dystrophy?
May Aung-Htut
Murdoch University
$50,699
Awardee: May Aung-Htut
Institution: Murdoch University
Award Amount: $50,699
Funding Period: February 1, 2019 - January 31, 2020
A novel RNA-based CRISPR strategy for treating congenital muscular dystrophy
Jamie Fitzgerald
Henry Ford Health System
$50,699
Awardee: Jamie Fitzgerald
Institution: Henry Ford Health System
Award Amount: $50,699
Funding Period: February 1, 2019 - January 31, 2020
Validation of pupil size as a biomarker for CDKL5 disorder: longitudinal assessment and relationship with disease severity
Tommaso Pizzorusso
University of Florence, Department NEUROFARBA
$51,382
Awardee: Tommaso Pizzorusso
Institution: University of Florence, Department NEUROFARBA
Award Amount: $51,382
Funding Period: February 1, 2019 - January 31, 2020
Anti-resorptive drugs in Fibrous Dysplasia of bone: studies on the effects of a RANKL inhibitor and Zoledronic Acid in a murine model of the disease by radiography, histology
Mara Riminucci
Sapienza University of Rome
$68,185
Awardee: Mara Riminucci
Institution: Sapienza University of Rome
Award Amount: $68,185
Funding Period: February 1, 2019 - January 31, 2020
Small molecule modulators of telomerase as novel treatments for dyskeratosis congenita
Suneet Agarwal
Boston Children’s Hospital
$100,373
Awardee: Suneet Agarwal
Institution: Boston Children's Hospital
Award Amount: $100,373
Funding Period: February 1, 2019 - January 31, 2020