A recent study explores adult-onset Sandhoff disease, revealing a motor neuron disease phenotype through patient-derived models. This research provides new mechanistic insights that could inform future therapeutic strategies.
adult onset sandhoff disease presenting with a motor neuron disease phenotype clinical and mechanistic insights from patient derived models
Original title: “Adult-onset Sandhoff disease presenting with a motor neuron disease phenotype: clinical and mechanistic insights from patient-derived models.”