Fragile X Syndrome: Supportive Treatment, Unmet Needs, and Paths to Novel Interventions and Disease-Targeted Therapies

Autor/in	Berry-Kravis, Elizabeth
Titel	Fragile X Syndrome: Supportive Treatment, Unmet Needs, and Paths to Novel Interventions and Disease-Targeted Therapies
Quelle	In: American Journal on Intellectual and Developmental Disabilities, 127 (2022) 2, S.90-94 (5 Seiten)Infoseite zur Zeitschrift PDF als Volltext Verfügbarkeit
Sprache	englisch
Dokumenttyp	gedruckt; online; Zeitschriftenaufsatz
ISSN	1944-7515
Schlagwörter	Genetic Disorders; Intellectual Disability; Autism; Pervasive Developmental Disorders; Neurological Impairments; Developmental Disabilities; Research; Intervention + Suchen Sie Ihr Suchwort? Intellect; Disability; Disabilities; Verstand; Behinderung; Autismus; Neurodegenerative Erkrankung; Entwicklungsstörung; Forschung
Abstract	Fragile X syndrome (FXS), as a monogenic cause of intellectual disability and autism spectrum disorder, has been one of the first neurodevelopmental disorders in which molecular and neuronal mechanisms of disease have been identified, leading to the concept of targeting the underlying disease to reverse symptoms. Translating findings in basic science and animal models to humans with FXS has proven difficult. These challenges have prompted the FXS field to organize to build interlocking projects to support initiatives to improve supportive care, make clinical research accessible to families, generate collaborative research on natural history and outcome measures, and create clinical trial consortia and novel trial designs. (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