2019 Research Grants Awarded

Dear Friends,

Thanks to your hard work and support, the National MPS Society is thrilled to announce the selection of $565,000 in new research grants through its annual research program. This year, the Society will fund a total of $822,500 that includes grant awards for both 2019, and the second-year distribution towards our 2018 grants. Therapies and cures will be discovered because of research programs like this – the Society is driven to help our children.

In April, the board of directors announced the availability of grants for
MPS I, MPS II, MPS III, MPS IV and general research, (open for all MPS and ML syndromes). Following a review of 28 received letters of intent, our Scientific Advisory Board reviewed requested proposals and recommended funding the following projects as the best research presented in each category.

This year’s grant selections are a true representation of an excellent global research program. Research will be funded to the best science in four countries. We are inspired to review excellent science submitted from new institutions. 

TWO YEAR GRANTS
 
$90,000 general grant
Dr. Ivan Conte
TIGEM
Pozzuoli Naples, ITALY
“Pharmacological pulsatile induction of autophagy as a novel therapeutic strategy for MPS-VII” 

$90,000 general grant/MPS II grant
Dr. Enrico Moro
University of Padova
Padova, ITALY
“Characterization of axon guidance defects in experimental in vitro and in vivo MPS II models” 

$90,000 general grant/MPS III grant
Dr. Nicollina Sorrentina
TIGEM
Pozzuoli Naples, ITALY
“Combining autophagy modulation and AAV-mediated gene delivery of lysosomal enzymes for a synergic treatment of CNS and somatic pathology in Mucopolysaccharidoses”

ONE YEAR GRANTS

$50,000 MPS IIIC grant
Dr. Liliana Matos
Ricardo Jorge INSA
Porto, PORTUGAL
“Exploring in vivo U1 snRNA splicing modulation as an alternative therapy for MPS IIIC”

$50,000 MPS IV grant
Dr. Maria Luz Couce
Santiago, IDIS
Santiago de Compostela, SPAIN
“Development a new lysosomal drug delivery system of elosulfase A for ERT of Morquio A”

$50,000 MPS general grant (Million Dollar Bike Ride)
University of Pennsylvania
Philadelphia, PA USA

$25,000 – multi-syndrome
Lysosomal Disease Network
University of Minnesota
Neuroimaging Core NIH Project

$15,000 – ML II/III alpha beta grant

ISMRD – partnership grant
USA

MPS II COLLABORATIVE RESEARCH GRANTS

$100,000 – MPS II grant
Dr. Patricia Dickson

Washington University, St. Louis, MO
Classification of IDS gene variants using saturation genome editing

$100,000 – MPS II grant
Dr. Richard Steet

Greenwood Genetic Center, Greenwood, SC
Functional characterization of novel IDS variants in cells and zebrafish

The National MPS Society’s professionally run and peer-reviewed research program continues to be recognized as a major funder of research, a supporter of young researchers and a collaborative effort with the NIH, family foundations, research institutions, and charities in the U.S. and globally.

We extend a huge THANK YOU to you and all the researchers who continue the fight for our children.

We look forward to soon announcing the research projects funded by the $100,000 raised in partnership with the University of Pennsylvania through the Million Dollar Bike Ride.

Sincerely,
Terri Klein, NPGC

President and CEO