Sandhoff disease
- Animals2
Trial sponsors
Masonic Cancer Center, University of Minnesota
1 trialPhase 2/3135 people
Latest
NeuroImmune Pharmacology and Therapeutics
Cinnamic acid, a natural plant compound, exhibits neuroprotection in a mouse model of Sandhoff disease via PPARα
Experiments in miceInterventions
Human Molecular Genetics
Intravenous administration of scAAV9-Hexb normalizes lifespan and prevents pathology in Sandhoff disease mice
Experiments in miceInterventions