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...
Scientific Research Grants
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 Institution: Telethon Institute of Genetic Medicine (TIGEM), Pozzouli, Italy Principal Investigator: Elvira De Leonibus, PhD, of TIGEM and the Italian National Research Council Duration: 24 months Start Date: July 1, 2021 Type of Sanfilippo studied: A...
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
Cure Sanfilippo Foundation has co-funded a research grant with Sanfilippo Children's Foundation (Australia) and H.A.N.D.S consortium, an international network of Type C families and groups. This grant is to Associate Professor Alessandro Fraldi at CEINGE - Advanced...
Investigating NDST1 inhibitors as target for substrate reduction therapy for Sanfilippo
Identifying a novel class of substrate reduction therapy drugs for the mucopolysaccharidoses that inhibit N-deacetylase/N-sulfo-transferase (NDST) Cure Sanfilippo Foundation has co-funded a one-year research project with Sanfilippo Children’s Foundation (Australia) to...
Exploring anti-fungal immunity and the airway microbiome in Sanfilippo syndrome (MPS III)
Cure Sanfilippo Foundation is funding a two-year project by Neta Shlezinger, PhD, at the Koret School of Veterinary Medicine in Israel, in collaboration with Dr. Annick Raas-Rothschild, Associate Professor and Director of Rare Diseases Institute at Sheba...
Creation of plasmid and efficacy study for MPS IIIC clinical trial
Cure Sanfilippo Foundation has funded New York-based Phoenix Nest, Inc. The funding supports creation of plasmid and the required in-vivo efficacy study in the Sanfilippo Type C (MPS IIIC) mouse model. These and other steps will enable the project to be evaluated by...
Re-Purposing FDA-Approved Inflammation Drug for Sanfilippo
Cure Sanfilippo Foundation is sponsoring an “Open-label Study of Anakinra in MPS III” clinical trial in collaboration with The Lundquist Institute (formerly LABiomed), and Sobi (Swedish Oprhan Biovitrium AB). This study is an open-label, single-arm, no placebo or...