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Orphan Designation: Treatment of mucopolysaccharidosis type I (MPS I)

Product Name : Iduronicrin genleukocel-T

Date Designated : March 19, 2018

Orphan Designation Status : Designated

Generic Name : Sleeping Beauty Transposon-Engineered Autologous Plasmablasts for Expression and Delivery of Alpha-L-Iduronidase

Trade Name : Iduronicrin Genleukocel-T (Iduroicel)

Approval Status : Not Approved