Cargando…

The STARS Phase 2 Study: A Randomized Controlled Trial of Gaboxadol in Angelman Syndrome

OBJECTIVE: To evaluate safety and tolerability and exploratory efficacy end points for gaboxadol (OV101) compared with placebo in individuals with Angelman syndrome (AS). METHODS: Gaboxadol is a highly selective orthosteric agonist that activates δ-subunit–containing extrasynaptic γ-aminobutyric aci...

Descripción completa

Detalles Bibliográficos
Autores principales:	Bird, Lynne M., Ochoa-Lubinoff, Cesar, Tan, Wen-Hann, Heimer, Gali, Melmed, Raun D., Rakhit, Amit, Visootsak, Jeannie, During, Matthew J., Holcroft, Christina, Burdine, Rebecca D., Kolevzon, Alexander, Thibert, Ronald L.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Lippincott Williams & Wilkins 2021
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8055330/ https://www.ncbi.nlm.nih.gov/pubmed/33443117 http://dx.doi.org/10.1212/WNL.0000000000011409

Ejemplares similares

Development of an adapted Clinical Global Impression scale for use in Angelman syndrome
por: Kolevzon, Alexander, et al.
Publicado: (2021)

Gaboxadol Normalizes Behavioral Abnormalities in a Mouse Model of Fragile X Syndrome
por: Cogram, Patricia, et al.
Publicado: (2019)

Angelman syndrome genotypes manifest varying degrees of clinical severity and developmental impairment
por: Keute, Marius, et al.
Publicado: (2020)

A Conceptual Model of Angelman Syndrome and Review of Relevant Clinical Outcomes Assessments (COAs)
por: Grieco, Joseph C., et al.
Publicado: (2018)

Healthcare burden among individuals with Angelman syndrome: Findings from the Angelman Syndrome Natural History Study
por: Khan, Nasreen, et al.
Publicado: (2019)

Delta power robustly predicts cognitive function in Angelman syndrome
por: Ostrowski, Lauren M., et al.
Publicado: (2021)

Electrophysiological Abnormalities in Angelman Syndrome Correlate With Symptom Severity
por: Hipp, Joerg F., et al.
Publicado: (2021)

Delta rhythmicity is a reliable EEG biomarker in Angelman syndrome: a parallel mouse and human analysis
por: Sidorov, Michael S., et al.
Publicado: (2017)

Erratum to: Delta rhythmicity is a reliable EEG biomarker in Angelman syndrome: a parallel mouse and human analysis
por: Sidorov, Michael S., et al.
Publicado: (2017)

Angelman syndrome: review of clinical and molecular aspects
por: Bird, Lynne M
Publicado: (2014)

Enabling endpoint development for interventional clinical trials in individuals with Angelman syndrome: a prospective, longitudinal, observational clinical study (FREESIAS)
por: Tjeertes, Jorrit, et al.
Publicado: (2023)

An observational study of pediatric healthcare burden in Angelman syndrome: results from a real-world study
por: Khan, Nasreen, et al.
Publicado: (2019)

Measuring What Matters to Individuals with Angelman Syndrome and Their Families: Development of a Patient-Centered Disease Concept Model
por: Willgoss, Tom, et al.
Publicado: (2020)

Preserved expressive language as a phenotypic determinant of Mosaic Angelman Syndrome
por: Carson, Robert P., et al.
Publicado: (2019)

Longitudinal EEG model detects antisense oligonucleotide treatment effect and increased UBE3A in Angelman syndrome
por: Spencer, Elizabeth R., et al.
Publicado: (2022)

Gaboxadol in Fragile X Syndrome: A 12-Week Randomized, Double-Blind, Parallel-Group, Phase 2a Study
por: Budimirovic, Dejan B., et al.
Publicado: (2021)

Angelman syndrome and anaesthetic considerations
por: Agarwal, Jyotsna, et al.
Publicado: (2017)

Abnormal coherence and sleep composition in children with Angelman syndrome: a retrospective EEG study
por: den Bakker, Hanna, et al.
Publicado: (2018)

Adeno-Associated Virus-Mediated Rescue of the Cognitive Defects in a Mouse Model for Angelman Syndrome
por: Daily, Jennifer L., et al.
Publicado: (2011)

Noninvasive screening tools for Down syndrome: a review
por: Smith, Meagan, et al.
Publicado: (2013)

High-voltage, diffuse delta rhythms coincide with wakeful consciousness and complexity in Angelman syndrome
por: Frohlich, Joel, et al.
Publicado: (2020)

High-voltage, diffuse delta rhythms coincide with wakeful consciousness and complexity in Angelman syndrome
por: Frohlich, Joel, et al.
Publicado: (2020)

Klinefelter syndrome and other sex chromosomal aneuploidies
por: Visootsak, Jeannie, et al.
Publicado: (2006)

A placebo-controlled trial of folic acid and betaine in identical twins with Angelman syndrome
por: Han, Julia, et al.
Publicado: (2019)

A multidisciplinary approach and consensus statement to establish standards of care for Angelman syndrome
por: Duis, Jessica, et al.
Publicado: (2022)

Angelman Syndrome and Angelman-like Syndromes Share the Same Calcium-Related Gene Signatures
por: Panov, Julia, et al.
Publicado: (2021)

Epilepsy in patients with Angelman syndrome
por: Fiumara, Agata, et al.
Publicado: (2010)

Neurodevelopmental Underpinnings of Angelman Syndrome
por: Li, Guohui, et al.
Publicado: (2014)

A web-based, patient driven registry for Angelman syndrome: the global Angelman syndrome registry
por: Napier, Kathryn R., et al.
Publicado: (2017)

A Male with Cooccurrence of Down Syndrome and Fragile X Syndrome
por: Anderson, Tovi, et al.
Publicado: (2013)

Behavior and neuropsychiatric manifestations in Angelman syndrome
por: Pelc, Karine, et al.
Publicado: (2008)

Epilepsy in Korean patients with Angelman syndrome
por: Park, Sung-Hee, et al.
Publicado: (2012)

Angelman Syndrome: A Case Report
por: Ashrafzadeh, Farah, et al.
Publicado: (2016)

Genotype–Phenotype Correlations in Angelman Syndrome
por: Yang, Lili, et al.
Publicado: (2021)

Stem cell models of Angelman syndrome
por: Camões dos Santos, João, et al.
Publicado: (2023)

Angelman syndrome: advancing the research frontier of neurodevelopmental disorders
por: Philpot, Benjamin D., et al.
Publicado: (2010)

A Case of False Negative NIPT for Down Syndrome-Lessons Learned
por: Smith, Meagan, et al.
Publicado: (2014)

Angelman syndrome and isovaleric acidemia: What is the link?
por: Lambrecht, Alix, et al.
Publicado: (2015)

Effect of epilepsy on autism symptoms in Angelman syndrome
por: Bakke, Kristin A., et al.
Publicado: (2018)

Pseudo Hypsarrhythmia: An Early Marker of Angelman Syndrome
por: Kasinathan, Ananthanarayanan, et al.
Publicado: (2019)

Cannot write session to /tmp/vufind_sessions/sess_k79dj59hb0mojuotdm248hept5