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MPS IIIA - Articles and news items

Abeona Therapeutics completes cohort in Sanfilippo syndrome trial

Industry news / 30 August 2016 / Niamh Louise Marriott, Digital Content Producer

The first-in-man clinical trial utilises a single injection of AAV gene therapy for patients with MPS IIIA (Sanfilippo syndrome type A), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death…