, health (Sleep Disorder Scale for Children, the Rett Syndrome Behavioural Questionnaire), parental health (12-item Short Form Health Survey) sociodemographic factors (parental employment and education) and quality of life (Quality of Life Inventory-Disability) for 210 individuals with Rett syndrome. ...
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med Read More Elevating Lives for More Than 30 Years For more than three decades, Acadia has been at the forefront of healthcare, elevating lives...
Anavex Life Sciences announced positive topline results from its Phase III AVATAR trial of Anavex 2-73 (blarcamesione) in adult female patients with Rett syndrome. February 1, 2022· 2 min read· Mark Terry Bio NC Clinical Catch-Up: January 10-14 Heading into the middle of January, ...
Yes - Fragile X syndrome; Rett syndrome; Infantile spasms New Molecular Entity Yes Highest Development Phases Preregistration Alzheimer's disease Phase II/III Fragile X syndrome; Rett syndrome; Unspecified Phase II Parkinson's disease Phase I Angelman syndrome No development reported Infantile...
Remains on track to provide regulatory update on registrational trial plans with NGN-401 for Rett syndrome in the first half… Read More Neurogene to Participate in Upcoming Conferences NEW YORK --(BUSINESS WIRE)--Feb. 24, 2025-- Neurogene Inc. (NASDAQ: NGNE), a clinical-stage company fou...
This review found two instruments to assess QoL in children and adolescents with DS and ten to assess HRQoL in the general pediatric population. Although, QI-Disability was designed specifically for children with intellectual disability such as Down syndrome, Rett syndrome, cerebral palsy or autism ...
The Company is in preparation for ANAVEX®2-73 for a Phase 2/3, placebo-controlled trial in Alzheimer’s disease as well as a Phase 2, placebo-controlled trial in Rett syndrome, for which the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for ANAVEX®...
which engages in the development of different therapeutics for the treatment of neurodegenerative and neurodevelopmental diseases. Its lead compound, ANAVEX2-73, is being developed to treat Alzheimer's and Parkinson's disease and central nervous system diseases, including Rett syndrome. The company was...
(CNS) diseases facing society today, includingAlzheimer’s disease,Parkinson’s disease, Schizophrenia and potentially other central nervous system diseases, including rare diseases, such asRett syndrome. We believe that targeting the upstream Sigma-1 receptor (SIGMAR1), through our proprietary orally ...
and both a Phase 2 and a Phase 3 study in adult patients and one Phase 2/3 study in pediatric patients with Rett syndrome. ANAVE2-73 is an orally available drug candidate designed to restore cellular homeostasis by targeting SIGMAR1 and muscarinic receptors. Preclinical studies demonstra...