MRIs Show Gene Therapy May Boost Brain Volume in Sanfilippo Type A

MRIs Show Gene Therapy May Boost Brain Volume in Sanfilippo Type A

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MRIs Show Gene Therapy May Boost Brain Volume in Sanfilippo Type A

A single high dose of ABO-102, Abeona Therapeutics’ experimental gene therapy, prevented disease-associated brain shrinkage in three young children with Sanfilippo syndrome type A for up to two years, according to updated interim data from the Transpher A Phase 1/2 clinical trial. “Brain volume loss is characteristic in children with MPS IIIA [Sanfilippo type A] and is associated with long-term cognitive and physical disability,” Vishwas Seshadri, PhD, Abeona’s head of research and clinical development, said in…

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