Ionis Pharmaceuticals’ investigational Angelman syndrome treatment ION582 demonstrated a favorable safety profile and lessened overall symptoms in patients participating…
Articles by Patricia Inacio, PhD
Ultragenyx Pharmaceutical has been cleared by the U.S. Food and Drug Administration to proceed to higher dosing of its…
A tailored behavioral intervention program led to longer sleep time, better sleep habits, and improved quality of life in children…
A history of reflux and night sweats during infancy may help identify children and adolescents with Angelman syndrome who…
The Foundation for Angelman Syndrome Therapeutics (FAST) has awarded a $500,000 grant to support the development of a less-invasive,…
The investigational gene therapy GTX-102 shows potential to lessen disease severity and improve function in children and…
A Phase 2 trial of NNZ-2591, Neuren Pharmaceuticals’ investigational oral therapy for Angelman syndrome, has started recruiting pediatric patients…
The U.S. Food and Drug Administration (FDA) has granted both orphan drug and rare pediatric disease status to ION582,…
A diet enriched with a nutritional formula supplemented with ketones — natural metabolites that arise from the breakdown of fat…
Epileptic seizures occur earlier and more frequently, leading to more hospitalizations, in children with Angelman syndrome due to a…