Awarded Grants
Awarded Grants
Galectin-1 protein therapy for the treatment for Laminin alpha2 related Congenital Muscular Dystrophy
Pam Van Ry
University of Nevada School of Medicine
35,000
Awardee: Pam Van Ry
Institution: University of Nevada School of Medicine
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
Unraveling the basis of white matter disease in mucopolysaccharidosis type I – a DTI study
Alena Svatkova
University of Minnesota
35,000
Awardee: Alena Svatkova
Institution: University of Minnesota
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
Targeting miR-155 as a therapy for mucolipidosis IV
Susan Slaugenhaupt
Massachusetts General Hospital
50,000
Awardee: Susan Slaugenhaupt
Institution: Massachusetts General Hospital
Award Amount: $50,000
Funding Period: January 1, 2015 - December 31, 2015
Identification of Molecular Targets for Pitt-Hopkins Syndrome Treatments
Benjamin Philpot
University of North Carolina, Chapel Hill
55,000
Awardee: Benjamin Philpot
Institution: University of North Carolina, Chapel Hill
Award Amount: $55,000
Funding Period: January 1, 2015 - December 31, 2015
Understanding TCF4 function in post-mitotic neuron synaptic plasticity
Daniel Marenda
Drexel University
55,000
Awardee: Daniel Marenda
Institution: Drexel University
Award Amount: $55,000
Funding Period: January 1, 2015 - December 31, 2015
Enhancing coagulation factor expression in skeletal muscle by augmenting the endogenous post-translational protein modification machinery: application to hemophilia gene therapy
Oscar Marcos-Contreras
The Children's Hospital of Philadelphia
35,000
Awardee: Oscar Marcos-Contreras
Institution: The Children's Hospital of Philadelphia
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
STAT3 in Pulmonary Lymphangioleiomyomatosis (LAM)
Vera Krymskaya
University of Pennsylvania
60,000
Awardee: Vera Krymskaya
Institution: University of Pennsylvania
Award Amount: $60,000
Funding Period: January 1, 2015 - December 31, 2015
Delineating the cause of gastrointestinal abnormalities in RASopathy disorders using human inducible pluripotent stem cells (iPSCs)
Maria Kontaridis
Beth Israel Deaconess Medical Center/Harvard Medical School
65,000
Awardee: Maria Kontaridis
Institution: Beth Israel Deaconess Medical Center/Harvard Medical School
Award Amount: $65,000
Funding Period: January 1, 2015 - December 31, 2015
CNS gene therapy to restore NPC1 protein expression in cerebellar Purkinje cells and delay or prevent disease onset in feline Niemann-Pick type C1 (NPC1) disease.
Brittney Gurda
University of Pennsylvania, School of Veterinary Medicine
35,000
Awardee: Brittney Gurda
Institution: University of Pennsylvania, School of Veterinary Medicine
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
Adeno-associated virus serotype 9-mediated gene therapy for Adrenomyeloneuropathy
Florian Eichler
Massachusetts General Hospital / Harvard Medical School
50,000
Awardee: Florian Eichler
Institution: Massachusetts General Hospital / Harvard Medical School
Award Amount: $50,000
Funding Period: January 1, 2015 - December 31, 2015
Pilot Study of the Efficacy and Safety of Sirolimus in the Treatment of Congenital Hyperinsulinism
Diva De Leon
Children's Hospital of Philadelphia
60,000
Awardee: Diva De Leon
Institution: Children's Hospital of Philadelphia
Award Amount: $60,000
Funding Period: January 1, 2015 - December 31, 2015
Reversal of epigenetic promoter silencing in Friedreich ataxia by histone deacetylase inhibitors
Yogesh Chutake
University of Oklahoma HSC
35,000
Awardee: Yogesh Chutake
Institution: University of Oklahoma HSC
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
The Molecular Mechanisms of PCSK9-induced LDLR Degradation
Chutikarn Butkinaree
Clinical Research Institute of Montreal
35,000
Awardee: Chutikarn Butkinaree
Institution: Clinical Research Institute of Montreal
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
Dual targeting of defective lipid and metal pathways in Niemann-Pick type C disease: an in vitro pilot study
Ashley Bush
The Florey Institute of Neuroscience and Mental Health
50,000
Awardee: Ashley Bush
Institution: The Florey Institute of Neuroscience and Mental Health
Award Amount: $50,000
Funding Period: January 1, 2015 - December 31, 2015
Quantitative description of the clinical course of Canavan disease
Annette Bley
University Medical Center Hamburg-Eppendorf, Dept of Pediatrics
35,000
Awardee: Annette Bley
Institution: University Medical Center Hamburg-Eppendorf, Dept of Pediatrics
Award Amount: $35,000
Funding Period: January 1, 2015 - December 31, 2015
Evaluation of PLK1 inhibitors in a pre-clinical LAM animal model
Aristotelis Astreinidis
Texas Tech University Health Sciences Center
60,000
Awardee: Aristotelis Astreinidis
Institution: Texas Tech University Health Sciences Center
Award Amount: $60,000
Funding Period: January 1, 2015 - December 31, 2015