Gene Therapy Attenuates Cognitive Decline in Sanfilippo Type B – Rare Disease Report
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Gene Therapy Attenuates Cognitive Decline in Sanfilippo Type B
Rare Disease Report A recent study published in Lancet Neurology found gene therapy, injected directly into the brain of 4 children with mucopolysaccharidosis type IIIB syndrome (MPS IIIB or Sanfilippo type B), resulted in sustained enzyme production and improvements in … |
