Mesenchymal Stem Cells for Sanfilippo Syndrome

Strengthening the rationale for the use of the “molecular tweezer” CLR01 in the treatment of Sanfilippo

Mar 31, 2021

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

Dec 30, 2020

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)

Nov 30, 2020

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...

Mesenchymal Stem Cells for Sanfilippo Syndrome

Engineering Mesenchymal Stem Cells (MSCS) to Produce Sulfamidase for Intrathecal Treatment of Sanfilippo Syndrome

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