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Abeona's Gene Therapy for Rare Sanfilippo Syndrome Shines in Early Trials

biospace.com
 2021-07-26

Cover picture for the articleAbeona Therapeutics’ shares popped 18.32% in pre-market trading after New York and Cleveland-based company announced magnetic resonance imaging (MRI) data from its Phase I/II Transpher A trial in Sanfilippo Syndrome Type A (MPS IIIA). MPS IIIA is a rare, fatal disease of lysosomal storage. It primarily affects the central nervous...

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