Details of the Drug

General Information of Drug (ID: DMY87RJ)

Drug Name

GTX-102

Indication

Disease Entry	ICD 11	Status	REF
Angelman syndrome	LD90.0	Phase 1/2	[1]
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Drug Type

Antisense oligonucleotide

Cross-matching ID

References

1	ClinicalTrials.gov (NCT04259281) A Phase 1/2 Open-label, Multiple-dose, Dose-escalating Clinical Trial of the Safety and Tolerability of GTX-102 in Pediatric Patients With Angelman Syndrome (AS). U.S.National Institutes of Health.