Search Orphan Drug Designations and Approvals
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Generic Name: | a Fab drug targeting the human TfR1 and conjugated to a PMO designed to bind to DMD exon 51 |
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Date Designated: | 03/21/2023 |
Orphan Designation: | Treatment of Duchenne Muscular Dystrophy |
Orphan Designation Status: | Designated |
FDA Orphan Approval Status: | Not FDA Approved for Orphan Indication |
Sponsor: |
Dyne Therapeutics, Inc. 1560 Trapelo Road Waltham, Massachusetts 02451 United States The sponsor address listed is the last reported by the sponsor to OOPD. |
*Data for the Date Designation Withdrawn or Revoked field are shown for designations withdrawn or revoked after 08/12/2013.
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