New Data on GTX-102 May Support Restart of Phase 1/2 Trial

New Data on GTX-102 May Support Restart of Phase 1/2 Trial

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New Data on GTX-102 May Support Restart of Phase 1/2 Trial

Further preliminary safety and efficacy data from a Phase 1/2 clinical trial into the investigational therapy GTX-102 in children and adolescents with Angelman syndrome may allow the study to resume patient enrollment and dosing. GeneTx Biotherapeutics and Ultragenyx , which are co-developing the treatment, shared these data in presentations at the recent Foundation for Angelman Syndrome Therapeutics (FAST) Global Summit. They support previous signs of treatment safety and potential efficacy. The companies placed the trial on hold last year after five…

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