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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: non-replicating recombinant adeno-associated virus vector containing 4 antisense sequences designed to target exon 2 in the human DMD gene under the control of U7 small nuclear non-coding RNA
Date Designated: 09/19/2017
Orphan Designation: Treatment of Duchenne muscular dystrophy
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
Nationwide Children's Hospital
Drug and Device Development Services
700 Children's Drive
Columbus, Ohio 43205
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*Exclusivity Protected Indications are shown for approvals from 01/01/2013 to the present.
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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