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About Sanfilippo
What is Sanfilippo?
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About Sanfilippo
What is Sanfilippo?
Patient Registry
Genetic Risk
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About Us
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About Sanfilippo
What is Sanfilippo?
Patient Registry
Genetic Risk
Sanfilippo Clinical Guidelines
Supporting Organisations
Research
Research Strategy
Global Research Roadmap
Solving Sanfilippo Symposium
Funding Program
Funded Projects
Generation of a novel preclinical animal model of Sanfilippo syndrome
PhD Scholarship: Using the brain in a dish model to screen drugs for Sanfilippo
Development of iminosugar-based pharmacological chaperones for Sanfilippo type B
Searching for "super active" enzymes for Sanfilippo Type A
Boosting lysosomal function and targeting neuroinflammation using a small compound for the treatment of Sanfilippo syndrome
Screening of potential therapeutics affecting lysosomal acidification in a fruit fly model of Sanfilippo
Insight into the role of heparan sulfate and dopamine as disease modifiers in Mucopolysaccharidosis type IIIA
Discovery and validation of translational biomarkers for Sanfilippo
Investigating the role of neuraminidase 1 in Sanfilippo
Optimising gene therapy for Sanfilippo Syndrome Type B
Targeting Autophagy in Sanfilippo Syndrome
PhD Scholarship: Molecular and bioinformatic studies of Alzheimer's and MPS III disease-linked genes in Zebrafish
PhD Scholarship: Investigating biomarkers of Sanfilippo Syndrome in accessible biological fluids
Development of nanoparticle drug delivery for Sanfilippo
Targeting the immune system as a treatment approach for Sanfilippo
Development and Testing of Sanfilippo Types A, B and C Zebrafish Models
A No Nonsense Approach to Sanfilippo Syndrome
Development of Pharmacological Chaperones for Sanfilippo type B
Further investigating the "molecular tweezer” CLR01 in the treatment of Sanfilippo Syndrome
Evaluation of SRT candidates for six Mucopolysaccharidoses
Therapeutic inhibition of a key inflammation pathway
Targeting Purinergic Signalling in the Brain to Reduce Inflammation in Sanfilippo
Creating a zebrafish model for Sanfilippo Type A
Investigating the effects of Sanfilippo Syndrome on the lungs
Designing "chaperone" drugs for Sanfilippo Type A
Targeting misfolded proteins for therapy for Sanfilippo Type C
Abeona Phase 1B trial of Gene Therapy program
PhD Scholarship - SAHMRI
PhD Scholarship - University of Adelaide
Investigating potential stem cell therapy for Sanfilippo Type B
Improving delivery of gene therapy to brain for Sanfilippo Type C
High throughput screening for new drugs to treat Sanfilippo
Investigating the role of brain lipids (fats) in Sanfilippo
Abeona Therapeutics Gene Therapy Program
Therapeutic strategies for the treatment of behavioural symptoms
Brain in a Dish Project: Development of cell models to search for new Sanfilippo treatments
Global Clinical Guidelines for Sanfilippo
Are there any long-term effects of being a Sanfilippo carrier?
Development of nanoparticle drug delivery for Sanfilippo
Evaluating potential drug therapies for Sanfilippo
Targeting autophagy as a therapeutic strategy for Sanfilippo
Therapy Avenues
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