Data from the Expansion Cohorts will be used to verify the GTX-102 dose and treatment regimen for the pivotal Phase 3 study. Angelman syndrome is a rare, neurogenetic disorder caused by ...
54 In summary, with the exception of the extreme phenotype of foetal alcohol syndrome, the evidence that maternally related cigarette and substance use and stress in pregnancy play a major causal role ...
Encoded Therapeutics Inc., a clinical-stage biotechnology company developing genetic medicines for severe central nervous system (CNS) disorders, announced today that it will present new preclinical ...
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced the pivotal Phase 3 study design following successful alignment with the U.S. Food and Drug Administration (FDA) on ION582, an ...