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O29: Results from a phase 1/2 open-label trial of GTX-102: An investigational antisense oligonucleotide for treatment of patients with Angelman syndrome

Author:

Kimberly Goodspeed,Tricia Cimms,Angel Chen,Wen-Hann Tan,Kenneth Myers,Elizabeth Berry-Kravis,Deike Weiss,Laurent Servais,Erick Sell

Publication:

Genetics in Medicine Open

Publisher:

Elsevier

Date:

2026

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