Pa­tient death in piv­otal gene ther­a­py tri­al rat­tles Sarep­ta part­ner al­ready fac­ing clin­i­cal hold

A child with mu­copolysac­cha­ri­do­sis type II­IA (MPS II­IA) has died in Lyso­gene’s piv­otal tri­al for its lead gene ther­a­py, four …

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