Funding Development of Nematode & Fly Disease Models and Screening Compounds

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...

Uniform gene delivery to the central nervous system (CNS) remains one of the most intractable challenges to the development of effective gene therapies for a wide range of neurological diseases, including MPS III (Sanfilippo Syndrome). While the brain microvasculature...

Cure Sanfilippo Foundation has again collaborated with Sanfilippo Children's Foundation of Australia to fund a new research project by TIGEM, investigating new therapeutic strategies for the treatment of the behavioral symptoms of Sanfilippo Syndrome (MPS III).TIGEM...