The Road to Personalized Therapies: Lessons Learned from Angelman Syndrome

Autor/in	Duis, Jessica
Titel	The Road to Personalized Therapies: Lessons Learned from Angelman Syndrome
Quelle	In: American Journal on Intellectual and Developmental Disabilities, 127 (2022) 2, S.95-98 (4 Seiten)Infoseite zur Zeitschrift PDF als Volltext Verfügbarkeit
Sprache	englisch
Dokumenttyp	gedruckt; online; Zeitschriftenaufsatz
ISSN	1944-7515
Schlagwörter	Neurological Impairments; Genetic Disorders; Expressive Language; Delayed Speech; Psychomotor Skills; Epilepsy; Sleep; Hyperactivity; Therapy; Outcomes of Treatment + Suchen Sie Ihr Suchwort? Neurodegenerative Erkrankung; Sprachverzögerung; Psychomotorische Aktivität; Epilepsie; Schlaf; Hyperaktivität; Therapie
Abstract	Angelman syndrome (AS) is a neurogenetic disorder characterized by delays including a severe expressive language delay, motor concerns, ataxia, epilepsy, sleep disturbances, gastrointestinal problems, and characteristic behaviors, including a happy demeanor, hyperactivity, and excitability. The syndrome is one of the first neurodevelopmental disorders with a clear trajectory towards meaningful treatment with approximately 20 companies actively developing targeted therapeutics for AS. Herein, we highlight the historical context of the road to therapeutics and some of the challenges in the field with the potential to impact the success of clinical trials for Angelman syndrome and also have relevance of other neurogenetic developmental disabilities. (As Provided).
Anmerkungen	American Association on Intellectual and Developmental Disabilities. P.O. Box 1897, Lawrence, KS 66044-1897. Tel: 785-843-1235; Fax: 785-843-1274; e-mail: AJMR@allenpress.com; Web site: https://meridian.allenpress.com/aaidd
Erfasst von	ERIC (Education Resources Information Center), Washington, DC
Update	2024/1/01