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U.S. Department of Health and Human Services

Search Orphan Drug Designations and Approvals

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Generic Name:  2’-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 (GYS1) pre mRNA
Date Designated:  10/08/2020
Orphan Designation:  treatment of Lafora disease
Orphan Designation Status:  Designated
FDA Orphan Approval Status:  Not FDA Approved for Orphan Indication
Ionis Pharmaceuticals, Inc.
2855 Gazelle Court
Carlsbad, California 92010
United States

The sponsor address listed is the last reported by the sponsor to OOPD.
*Exclusivity Protected Indications are shown for approvals from Jan. 1, 2013, to the present.