Million Dollar Bike Ride 2019 Pilot Grant Awardees

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MDBR, FOP
MDBR, FOP, 2019
Eludicating the role of marrow-resident myeloid cells in FOP
MDBR, FOP
MDBR, FOP, 2019

Shailesh Agarwal

Brigham and Women's Hospital, Harvard University

$40,208

MDBR, FOP
MDBR, FOP, 2019
MDBR, MPS Gene Spotlight, MPS
MDBR, MPS Gene Spotlight, MPS, 2019
Attention-deficit and hyperactivity links to the brain connectivity of MPS I subjects: multimodal MRI study
MDBR, MPS Gene Spotlight, MPS
MDBR, MPS Gene Spotlight, MPS, 2019

Igor Nestrasil

University of Minnesota

$61,589

MDBR, MPS Gene Spotlight, MPS
MDBR, MPS Gene Spotlight, MPS, 2019
MDBR, IBM
MDBR, IBM, 2019
DEVELOPMENT OF A CHEMOKINE TARGETED THERAPY FOR INCLUSION BODY MYOSITIS
MDBR, IBM
MDBR, IBM, 2019

Steven Greenberg

Brigham and Women's Hospital

$102,430

MDBR, IBM
MDBR, IBM, 2019
MDBR, GLUT1
MDBR, Glut1, 2019
Defining the cellular site of action of the Glut1 protein in Glucose Transporter 1 deficiency syndrome.
MDBR, GLUT1
MDBR, Glut1, 2019

Umrao Monani

Columbia University

$46,858

MDBR, GLUT1
MDBR, Glut1, 2019
MDBR, LAM
MDBR, LAM, 2019
Targeting mitochondrial oxidative metabolism in LAM using a first-in-class lipoate analog
MDBR, LAM
MDBR, LAM, 2019

Carmen Priolo

Brigham and Women's Hospital, Harvard Medical School

$72,704

MDBR, LAM
MDBR, LAM, 2019
MDBR, LGDALMI
MDBR, LGDALMI, 2019
Engineered microvessels derived from human lymphatic endothelium as an in vitro model for drug repurposing for lymphatic malformations
MDBR, LGDALMI
MDBR, LGDALMI, 2019

Julie Blatt

UNC Chapel Hill

$62,861

MDBR, LGDALMI
MDBR, LGDALMI, 2019
MDBR, ML4
MDBR, ML4, 2019
A Prospective Natural History Study of Mucolipidosis type 4
MDBR, ML4
MDBR, ML4, 2019

Albert Misko

Massachusetts General Hospital

$78,538

MDBR, ML4
MDBR, ML4, 2019
MDBR, MPS
MDBR, MPS, 2019
A comparative and quantitative approach to study lysosomal dysfunction in Mucopolysaccharidoses diseases
MDBR, MPS
MDBR, MPS, 2019

Andrea Ballabio

Fondazione Telethon, TIGEM

$64,063

MDBR, MPS
MDBR, MPS, 2019
MDBR, MSUD
MDBR, MSUD, 2019
Can we improve neurologic and psychiatric outcomes in MSUD? Studies in a new mouse model
MDBR, MSUD
MDBR, MSUD, 2019

Rebecca Ahrens-Nicklas

The Children's Hospital of Philadelphia / The University of Pennsylvania

$44,037

MDBR, MSUD
MDBR, MSUD, 2019
MDBR, NBIA(BPAN), NBIA, BPAN
MDBR, NBIA, BPAN, NBIABPAN, 2019
Development of novel human stem cell models of Beta-Propeller Protein- Associated Neurodegeneration (BPAN) for disease modelling and drug screening
MDBR, NBIA(BPAN), NBIA, BPAN
MDBR, NBIA, BPAN, NBIABPAN, 2019

Paul Lockhart

Murdoch Children's Research Institute

$61,245

MDBR, NBIA(BPAN), NBIA, BPAN
MDBR, NBIA, BPAN, NBIABPAN, 2019