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Njc Smith

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Safety, Tolerability, and Preliminary Evidence of Biopotency in Transpher B, a Multicenter, Singledose, Phase 1/2 Clinical Trial of ABO-101 Gene Therapy for Sanfilippo Syndrome Type B (Mucopolysaccharidosis IIIB) (5175)

2020 Neurology
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A Phase 2/3 Study of the Efficacy and Safety of Ex Vivo Gene Therapy with Lenti-DTM Lentiviral Vector for the Treatment of Cerebral Adrenoleukodystrophy

2016
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