Antisense Oligonucleotides
Ultragenyx’s GTX-102 Shows Promising Results in Mid-Stage Angelman Syndrome Study, Pivotal Study on the Horizon
Ultragenyx, GTX-102, Angelman Syndrome, Mid-Stage Study, Pivotal Study, Antisense Oligonucleotide, Cognitive Improvement, Behavioral Improvement, Sleep Improvement, Gross Motor Improvement
Groundbreaking Data Revealed for Genetic Epilepsy Drug STK-001 by Stoke Therapeutics, Exhilarating Investors
Stoke Therapeutics, STK-001, Genetic Epilepsy Drug, Dravet Syndrome, Antisense Oligonucleotide, Seizure Reduction, Positive Clinical Trial Data, Investor Enthusiasm