Sanfilippo Syndrome News and Research

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Rare Disease Day 2021: Raising awareness of childhood dementia

Rare Disease Day 2021: Raising awareness of childhood dementia

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children

A new combination therapy for treating rare, fatal Sanfilippo syndrome in children

Study provides new insights into an untreatable form of childhood-onset dementia

Study provides new insights into an untreatable form of childhood-onset dementia

New brain cell models created to evaluate therapies for Sanfilippo C syndrome

New brain cell models created to evaluate therapies for Sanfilippo C syndrome

Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Gene therapy is key to addressing Sanfilippo Syndrome, say Ohio scientists and clinicians

Research finding opens door to potential treatment for MPS IIIB

Research finding opens door to potential treatment for MPS IIIB

Abeona Therapeutics obtains FDA Orphan Drug Designations for Sanfilippo Syndrome therapies

Abeona Therapeutics obtains FDA Orphan Drug Designations for Sanfilippo Syndrome therapies

Study highlights benefits of using multiple injections to treat Sanfilippo syndrome type III B

Study highlights benefits of using multiple injections to treat Sanfilippo syndrome type III B

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

Gene therapy developed by UAB cures pediatric Sanfilippo Syndrome A in animal models

Renaissance in drug development for rare diseases

Renaissance in drug development for rare diseases

LYSOGENE launches the first gene therapy clinical trials in Sanfilippo disease

LYSOGENE launches the first gene therapy clinical trials in Sanfilippo disease

Investigators receive NIH grant to help move gene therapy for MPS IIIB toward clinical trials

Investigators receive NIH grant to help move gene therapy for MPS IIIB toward clinical trials

Sanfilippo syndrome compromises blood-brain barrier, leads to brain damage

Sanfilippo syndrome compromises blood-brain barrier, leads to brain damage

New understanding of Sly Syndrome

New understanding of Sly Syndrome