Chief investigator Professor Kim Hemsley of Flinders University.The Sanfilippo Children’s Foundation and Cure Sanfilippo Foundation have awarded an Incubator Grant to Professor Kim Hemsley at Flinders University. Professor Hemsley and her collaborators, Dr Siti...
Scientific Research Grants
Combination of stem cell transplantation and cathepsin B inhibitors for treatment of Sanfilippo
Chief investigator Alexey Pshezhetsky, PhD, of Centre Hospitalier Universitaire Sainte-Justine, Universite de MontrealCure Sanfilippo Foundation and Sanfilippo Children’s Foundation (Australia) have awarded a Translational Grant to Professor Alexey Pshezhetsky at the...
Blood-based biomarkers for Sanfilippo syndrome progression and treatment
Grant Summary Institution: University of California, Los Angeles Primary Investigator: Gal Bitan, Ph.D./Professor of Neurology Duration: 24 months Start Date: September 2021 Type of Sanfilippo studied: Types A, B, C, and D Types of Sanfilippo that could benefit: Types...
Identifying Disease Modifiers in Drosophila Models of Sanfilippo (MPS IIIA)
Grant Summary Institution: Clemson University, South Carolina, U.S. Primary Investigator: Trudy Mackay, PhD, Director, Clemson Center for Human Genetics and Self Family Endowed Chair of Human Genetics Duration: 24 months Start Date: January 2022 Type of Sanfilippo...
Discovery & validation of translational biomarkers for Sanfilippo childhood dementia
Page reviewed by: Dr. Cara O'Neill, FAAP and Jan Kaslin, PhD Page last updated: February 18, 2022 For web accessibility options: Click/tap the floating blue icon on the right. Grant Summary Institution: Monash University, Victoria, Australia Primary Investigator: Jan...
Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Sanfilippo
Grant Summary Institution: University of Naples Federico II, Naples, Italy Investigator: Luigi Michele Pavone, Associate Professor of Biochemistry, Department of Molecular Medicine and Medical Biotechnology, Medical School, University of Naples Federico II Duration:...
Exploring the role of heparan sulfate and dopamine as disease modifiers in Sanfilippo
Grant Summary Grant Title:Insight into the role of heparan sulfate and dopamine as disease modifiers inMucopolysaccharidosis type IIIA Institution: Telethon Institute of Genetic Medicine (TIGEM), Pozzouli, Italy Principal Investigator: Elvira De Leonibus, PhD, of...
Newborn screening pilot ScreenPlus that includes Sanfilippo Type A and B
When children with a rare disease are diagnosed at birth, they have the greatest opportunity to receive timely, approved treatments or participate in clinical trials that can provide them better quality of life and less pain and suffering. Early diagnosis and...
Strengthening the rationale for the use of the “molecular tweezer” CLR01 in the treatment of Sanfilippo
Page last updated: Aug. 21, 2024 Page reviewed by: Dr. Cara O'Neill, FAAP For web accessibility options: Click/tap the floating blue icon on the right.Cure Sanfilippo Foundation has co-funded a research grant with Sanfilippo Children's Foundation (Australia) and...