Submitted by Anonymous (not verified) on 10 June 2025 - 7:32
Orphan designation: Synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts Treatment of Angelman syndrome, 09/12/2020 Positive
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Orphan designation: Synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts Treatment of Angelman syndrome, 09/12/2020 Positive