2015

2015 Research Grants:

Targeting mTORCI and autophagy pathways to rescue the skeletal phenotype in MPS mouse models

Dr. Carmine Settembre
Telethon Institute of Genetics and Medicine of Fondazione Telethon
Pozzuoli, Italy

Validation of small molecule therapeutic leads for treatment of MPS I disease

Dr. Allison R. Kermode, Professor
Simon Fraser University
Burnaby, BC Canada

AAV2/8 medicated expression of modified sulphamidase: Liver targeting for improved secretion and brain deliver. Pre-clinical study in the Huntaway dog

Dr. Kim Hemsley, Dr. Alessandro Fraldi, and Professor Robert D. Jolly
Lysosomal Diseases Research Unit
Adelaide, SA, Australia

Creating new tools for understanding skeletal disease in MPS IVA

Dr. Ainslie Derrick-Roberts
Genetics and Molecular Pathology
North Adelaide, SA, Australia

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

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The National MPS Society exists to cure, support and advocate for MPS and ML.

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