MDBR 2015

Click the image below to see the awesome 2015 MDBR video!

Click Image to see the 2015 MDBR video!

Click Image to see the 2015 MDBR video!

MDBR2015 infographic (hi-res)MDBRFlyer2015team

 

 

 

 

 

 

 

 

2015 MDBR Grant Awards

The 2nd Annual Million Dollar Bike Ride on Saturday, May 9, 2015 was a huge success. The Orphan Disease Center welcomed 600 cyclists and volunteers, more than a dozen neighborhood restaurants, and several supporting sponsors and community partners. Twenty three disease teams from 29 states (plus Canada, UK, Chile and the Netherlands!) rode a combined total of nearly 18,000 miles in the name of rare disease research. In just two years, the Million Dollar Bike Ride has raised over $2 million, 100% of which goes directly towards pilot research grants (with no administrative cut taken out).

This year, we are awarding 26 grants, for 16 rare diseases, to 21 institutions worldwide. The ODC is proud to announce the awardees of the 2015 MDBR Rare Disease Research Grants, listed below:

Team Stop ALD
Florian Eichler, MD
Massachusetts General Hospital, Harvard Medical School
Adrenoleukodystrophy pilot for $50,000
Optimizing Delivery and Endpoints of an AAV9-mediated Gene Therapy Trial in AMN

Team Bronchiolitis Obliterans
Martin Rosewich, MD
Goethe-University Frankfurt/M, Germany
Bronchiolitis Obliterans pilot for $50,500
Role of microRNAs in the pathophysiology of bronchiolitis obliterans

Team Bronchiolitis Obliterans
Stephen Stick, MB, BChir, PhD
Princess Margaret Hospital for Children, Perth, Western Australia
Bronchiolitis Obliterans pilot for $50,500
Epithelial-mesenchymal transition in pediatric airway epithelial cells following infection with bronchiolitis obliterans associated adenoviruses.

Team Castleman Disease Network
Minji Byun, PhD
Washington University School of Medicine in St. Louis
Castleman Disease pilot for $63,000
Whole genome sequencing based discovery of germline mutations underlying idiopathic multicentric Castleman disease

Team Emily’s Entourage
Steven Rowe, MD, MSPH
University of Alabama at Birmingham
CFTR Nonsense Mutations pilot for $51,500
An open label N of 1 study to evaluate the safety and efficacy of long-term treatment with Ivacaftor in combination with ATALUREN (PTC124) in Subjects with Nonsense Mutation Cystic Fibrosis

Team Emily’s Entourage
Christine Bear, PhD
SickKids Hospital-Toronto
CFTR Nonsense Mutations pilot for $51,500
Evaluating “exon‐skipping” as a strategy to repair the CFTR nonsense mutation

Team Movin’ for Mallory
Garry Cutting, MD
Johns Hopkins University School of Medicine
Cystic Fibrosis pilot for $69,000
Systematic Evaluation of a Cluster of Nonsense Mutations in Exon 22 of CFTR to Inform Treatment

Team Raring to Go for CHI
Mark Dunne, BSc, PhD
University of Manchester
Congenital Hyperinsulinism pilot for $71,000
Towards Precision Medicine in the Treatment of Congenital Hyperinsulinism in Infancy

Team Bike to End Duchenne
Lisa Maves, PhD
Seattle Children’s Research Institute
Duchenne Muscular Dystrophy pilot for $49,000
A zebrafish platform for DMD drug-combination testing

Team Fibrous Dysplasia
J. Silvio Gutkind, PhD
University of California San Diego, Moores Cancer Center
Fibrous Dysplasia pilot for $58,500
Development of next-gen animal models for fibrous dysplasia (FD)/McCune-Albright syndrome (MAS)

Team Fibrous Dysplasia
Mara Riminucci
Sapienza University of Rome
Fibrous Dysplasia pilot for $58,500
Mouse models of Fibrous Dysplasia (FD) as a tool for developing rational and effective therapies

Team Ride for LGDA & Team LMI
Maxim Itkin, MD
University of Pennsylvania
GLA/GSD pilot for $101,000
Dynamic Contrast Enhanced MR Lymphangiogram Imaging of Lymphatic Anomalies (LA)

Team Ride for LGDA & Team LMI
Joseph Rutkowski, PhD
UT Southwestern Medical Center
GLA/GSD pilot for $51,000
Inducible lymphatic hyperplasia to drive chylous accumulation mimicking lymphangiomatosis

Team Ride for LGDA & Team LMI
David Cormode, PhD
University of Pennsylvania
GLA/GSD pilot for $51,000
Nano-magnetic oils for the study of intestinal lymphatics in lymphangiomatosis

Team LAM Foundation Easy Breathers
David Kwiatkowski, MD, PhD
Brigham and Women’s Hospital
Lymphangioleiomyomatosis pilot for $50,500
TSC2 mutation analysis as a biomarker for lymphangioleiomyomatosis (LAM)

Team LAM Foundation Easy Breathers
Norbert Perrimon, PhD
Harvard Medical School
Lymphangioleiomyomatosis pilot for $50,500
An shRNA based screening method for TSC synthetic lethal screens

Team Cure ML4
Kartik Venkatachalam
University of Texas, School of Medicine at Houston
Mucolipidosis Type IV pilot for $53,000
Mechanisms of Neurodevelopmental Defects in Mucolipidosis type IV (MLIV)

Team MPS
Charles Vite, DVM, PhD, B.S.
University of Pennsylvania
Mucopolysaccharidoses pilot for $50,500
Natural history study and biomarker development in the large animal model of mucolipidosis II

Team MPS
Igor Nestrasil, MD
University of Minnesota
Mucopolysaccharidoses pilot for $50,500
Microstructural and functional MRI signatures in patients with MPS I

Team NPC
Andrew Lieberman, MD, PhD
University of Michigan
Niemann-Pick Disease Type C pilot for $53,500
Characterization of NPC1-I1061T proteostasis machinery for development of NPC therapeutics

Team NPC
Charles Vite, DVM, PhD, B.S.
University of Pennsylvania
Niemann-Pick Disease Type C pilot for $53,500
Targeted AAV gene therapy for vertical supranuclear gaze palsy and dysphagia

Team Pitt Hopkins Pedalers
Tonis Timmusk, PhD
Tallinn University of Technology
Pitt Hopkins pilot for $50,000
Regulation of TCF4 transcriptional activity in neurons

Team Pitt Hopkins Pedalers
Brady Maher, PhD
Lieber Institute for Brain Development, Johns Hopkins School of Medicine
Pitt Hopkins pilot for $50,000
Exploring the impact of a TCF4 mutation on the physiology of inhibitory neurons of the prefrontal cortex

Team RASopathies Network Riders
Giuseppe Zampino
Università Cattolica Sacro Cuore
RASopathies pilot for $53,000
Pain in RASopathies: new investigative techniques and treatments

Team NTSAD
Marlene Jacobson, PhD
Temple University School of Pharmacy
Tay-Sachs & Allied Diseases pilot for $43,000
Patient-Derived Phenotypic Assay to Discover Treatments for Tay-Sachs Disease

Team UCD
Ljubica Caldovic, PhD
Children’s National Health System
Urea Cycle Disorders pilot for $43,000
CNS Protection from Hyperammonemia

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Check out the 2015 MDBR jersey!

Jersey available for fundraising cyclists only.

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2015 Sponsors

 

Mike Casias, who cycled for the Movin’ for Mallory Team, won our raffle for a brand new bicycle provided by Specialized.

Mike Casias, who cycled for the Movin’ for Mallory Team, won our raffle for a brand new bicycle provided by Specialized.