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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals
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Generic Name: exon 53 specific phosphorothioate oligonucleotide
Date Designated: 01/23/2013
Orphan Designation: treatment of Duchenne Muscular Dystrophy patients bearing mutations that can be corrected by skipping exon 53
Orphan Designation Status: Designated
FDA Orphan Approval Status: Not FDA Approved for Orphan Indication
BioMarin Pharmaceutical, Inc.
105 Digital Drive
Novato, California 94949
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*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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