Patricia Inacio, PhD, science writer —

Patricia holds her PhD in cell biology from the University Nova de Lisboa, Portugal, and has served as an author on several research projects and fellowships, as well as major grant applications for European agencies. She also served as a PhD student research assistant in the Department of Microbiology & Immunology, Columbia University, New York, for which she was awarded a Luso-American Development Foundation (FLAD) fellowship.

Articles by Patricia Inacio

Trial: Gene Therapy GTX-102 Shows Promise in Angelman Children

The investigational gene therapy GTX-102 shows potential to lessen disease severity and improve function in children and adolescents with Angelman syndrome, according to interim data from a Phase 1/2 clinical trial. Six-month follow-up data for 11 patients showed that treatment with GTX-102 resulted in improvements across exploratory…

Phase 2 Trial of NNZ-2591 for Angelman Opens in Australia

A Phase 2 trial of NNZ-2591, Neuren Pharmaceuticals’ investigational oral therapy for Angelman syndrome, has started recruiting pediatric patients in Australia. The Australian study — which was also given regulatory clearance in the U.S. — will be conducted at three hospitals in that country: one each in Queensland, New…

FDA Acts to Support Development of Ionis Therapy ION582

The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to ION582, Ionis Pharmaceutical’s investigational treatment for Angelman syndrome. The announcement of the new FDA designations — both aimed at encouraging the therapy’s development — follows the launch of the…

Study Links Missing Gene to Severe Seizures in Angelman Children

Epileptic seizures occur earlier and more frequently, leading to more hospitalizations, in children with Angelman syndrome due to a missing UBE3A gene, according to a retrospective analysis of a natural history study. The study, “Clinical Characterization of Epilepsy in Children With Angelman Syndrome,” was published in the journal…

Trial for GTX-102 to Open Enrollment Soon in the UK

The United Kingdom’s Medicines Healthcare Products Regulatory Agency (MHRA) has authorized a Phase 1/2 clinical trial to test the investigational therapy GTX-102 in children and adolescents with Angelman syndrome. The open-label KIK-AS trial (NCT04259281), which also has been given a green light in Canada, is expected to open enrollment in the…