Research Grants

Grants and fellowships from the National MPS Society are provided to qualified medical researchers for the purpose of promoting medical research in the fields of MPS and ML. Each April, the MPS Society announces the grants offered, and funding begins October 1. Occasionally a second grant offering is made throughout the year.

All applications are reviewed and ranked by members of the National MPS Society Scientific Advisory Board. The final decision of award recipients is determined by the board of directors of the National MPS Society. Funding for the grants comes from the annual National MPS Society 5K walk/runs in addition to donations and fundraisers. We have awarded more than $7 million in grant funding since 2001.

Notice of the current year grant offerings and application will appear on the website. To receive the current application by e-mail, please contact us.


The National MPS Society has allocated $335,000 in grant funding for 2016 which includes the second year funding for grants awarded in 2015. We received many letters of intent from researchers around the world for research grants. After reviewing those letters, our Scientific Advisory Board review committee requested full grant proposals from researchers. Two new grants were chosen for MPS IVA and for MPS VII. Additionally, $100,000 of funds raised were through the Million Dollar Bike Ride effort, and these funds have been allocated through the University of Pennsylvania for MPS research grants.

The Board of Directors allocated $30,000 through the Fundraising Directive Program. The family who raised these funds requested work be continued with Dr. Haiyan Fu of the Research Institute at Nationwide Children’s Hospital for gene therapy approach for advanced MPS II via AAV9 vectors.

We also provided $25,000 to support the Lysosomal Disease Network’s NIH grant research goals. The funding is designed for the Neuroimaging Core, which will benefit the four MPS projects. The MPS Society also provided $5,000 in partnership with the Ryan Foundation for an MPS I project.

In the fourth quarter of 2016, we raised an additional $150,000 in research funds to be awarded for two new projects in the first quarter of 2017. $135,000 will be awarded to Dr. Charbel Moussa of Georgetown University for an MPS IIIA drug repurposing project and $30,000 will be awarded in a partnership grant with the ISMRD (International Society for Mannosidosis and Related Diseases) for novel ML gene therapy research with Dr. Stephen Gray at UNC Hospital in partnership with Dr. Charles Vite at the University of Pennsylvania.

2016 Research Grants:

AAV2/8-mediated expression of modified sulphamidase, liver targeting for improved secretion and brain delivery in IIIA Huntaway dog

Dr. Kim Hemsley
South Australia Health and Medical Research Institute
Adelaide, South Australia

Gene Therapy for MPS IVA

Dr. Kazuki Sawamoto, Dr. Shunji Tomatsu
Nemours/Alfred I. duPont Hospital for Children
Wilmington, DE

Therapeutic Targeting of Wnt/β-Catenin Signaling to Improve Bone Formation in MPS VII

Dr. Lachlan J. Smith
University of Pennsylvania
Philadelphia, PA

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


2014 Research Grants:

The National MPS Society allocated $509,000 in grant funding for 2014 which includes the second year funding for grants awarded in 2013, the unspent MPS III funds from 2012 and the 2014 grants. The funding we provide is critical as we move forward with our mission to find cures for MPS and ML. We received 20 letters of intent from researchers around the world for the General, MPS II, MPS IVA and MPS VI grants. After reviewing those letters, our Scientific Advisory Board review committee requested full grant proposals from eight researchers.

The board of directors allocated $100,000 to Abeona Therapeutics which has licensed the MPS IIIA and MPS IIIB gene therapy technology from Nationwide Children’s Hospital. Funds already raised by Abeona have been funneled to Nationwide for MPS III drug manufacturing and preclinical research plus two INDs (investigational new drug applications). The financial distribution from the Society will help move the clinical trial forward.

We also provided $25,000 to support the Lysosomal Disease Network’s NIH grant research goals. The funding is designed for the Neuroimaging Core, which will benefit the four MPS projects. An additional $8,000 was offered for an ML grant in partnership with ISMRD (International Society for Mannosidosis and Related Diseases). A $10,000 partnership grant with the Ryan Foundation funds the University of MN project “Longitudinal Studies of Brain Structure and Function in MPS Disorders.” We also provide funding for post-doctoral fellows to attend the American Society and Gene and Cell Therapy conference.

The role of angiotensin II-mediated inflammation in MPS I vascular disease: a study of pathophysiologic mechanism and evaluation of angiotensin receptor blockade therapy

Moin Vera, MD, PhD
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, CA

AAV mediated gene transfer to the CNS for MPS II

Scott McIvor, PhD
University of Minnesota
Minneapolis, MN

Overcoming limitations inherent in sulfamidase to improve MPS IIIA gene therapy

Beverly Davidson, PhD
The Children’s Hospital of Philadelphia

Pentosan Polysulfate and GAGs in MPS

Icahn School of Medicine at Mount Sinai
New York, NY


2013 Research Grants:

The National MPS Society allocated $530,000 in grant funding for 2013, which includes the second year funding for grants awarded in 2012, plus the 2013 grants. The funding we provide is critical as we move forward with our mission to find cures for MPS and ML. We received 48 letters of intent from researchers around the world for the six grants offered in 2013. After reviewing those letters, our Scientific Advisory Board review committee requested full grant proposals from 13 researchers.

The MPS Society will also fund $25,000 to support the Lysosomal Disease Network’s NIH grant research goals. The funding is designed for the Neuroimaging Core, which will benefit the four MPS projects. An additional $20,000 will be offered for an ML grant in partnership with ISMRD (International Society for Mannosidosis and Related Diseases). A $10,000 partnership grant with the Ryan Foundation funded the University of MN project “Longitudinal Studies of Brain Structure and Function in MPS Disorders.” The National MPS Society also provides funding for post-doctoral fellows to attend the Gordon Conference on lysosomal diseases.

Pathogenesis of Bone Disease in Mucopolysaccharidosis Disorders

two years @ $30,000 each year
Lachlan Smith, PhD
University of Pennsylvania
Philadelphia, PA

Adjunctive therapy for Hurler syndrome.

Richard Steet, PhD
University of Georgia
Athens, GA

AND

Dr. Dwight Koeberl
Duke University
Durham, NC

Development of pharmacological chaperone therapy for MPS II.

Vito Ferro, PhD
University of Queensland
Brisbane, Queensland
Australia

Delivery of sulfamidase to the brain.

Jeffrey Esko, PhD
University of California, San Diego
La Jolla, CA

Manifestations of Cardiovascular Disease in Morquio A: Evaluation, Assessment, and Therapy

Adriana Montano, PhD
St. Louis University
St. Louis, MO

AND

Raymond Wang, M.D.
CHOC Children’s Hospital
Orange, CA


2012 Research Grants:

The National MPS Society awarded $547,000 in grant funding for 2012 which includes the second year funding for grants awarded in 2011 plus the 2012 grants. The funding we provide is critical as we move forward with our mission to find cures for MPS and ML. We received 16 letters of intent from researchers around the world for the three grants offered in 2012. After reviewing those letters, our Scientific Advisory Board review committee requested full grant proposals from seven researchers.

We also provided $25,000 to support the Lysosomal Disease Network’s NIH grant research goals. The funding is designed for the Neuroimaging Core, which will benefit the four MPS projects. An additional $15,000 has been allocated for a mucolipidosis partnership grant with the Gandhi Foundation to Dr. Sara Cathey at Greenwood Genetics Center, “PTC 124 for nonsense mutation suppression in ML II and III cultured fibroblasts.” A $10,000 partnership grant with the Ryan Foundation funded the University of MN project “Brain Structure and Function in Developmentally Normal Children Ages 4-7.” The National MPS Society also provides funding for post-doctoral fellows to attend scientific meetings, such as the American Society of Gene and Cell Therapy.

Induced-neuronal (iN) cells as tools to study the pathogenesis of neurological manifestations in MPS-II

Gustavo H.B. Maegawa, MD, PhD
Johns Hopkins School of Medicine, Department of Pediatrics
Baltimore, MD

Development of Long Circulating Enzyme Replacement Therapy for MPS IVA.

Shunji Tomatsu, MD, PhD
Nemours Children’s Clinic – Delaware Valley of the Nemours Foundation
Wilmington, DE

Dr. Brian Bigger
Stem Cell & Neurotherapies Group
Manchester, UK
“Evaluation of high dose genistein aglycone in the treatment of  mucopolysaccharide disease
types IIIA, B and C.”


Previous Grant Awards

2011

2010

2009

2008

2007

2006

2005

2004

2003

2002

2001

Our Mission

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

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