Translational research for the development of treatment of patients with neurogenetic diseases: An important step for Angelman syndrome
Author:
Publisher
Elsevier BV
Subject
Neurology (clinical),General Medicine,Pediatrics, Perinatology and Child Health
Reference5 articles.
1. UBE3A reinstatement as a disease-modifying therapy for Angelman syndrome;Elgersma;Dev. Med. Child Neurol.,2021
2. Decreased tonic inhibition in cerebellar granule cells causes motor dysfunction in a mouse model of Angelman syndrome;Egawa;Sci. Transl. Med.,2012
3. Disruption of the LTD dialogue between the cerebellum and the cortex in Angelman syndrome model: a timing hypothesis;Cheron;Front. Syst. Neurosci.,2014
4. Gaboxadol in Angelman syndrome: a double-blind, parallel-group, randomized placebo-controlled phase 3 study;Keary;Eur. J. Paediatr. Neurol.,2023
5. Individuals with lived experience of disability should participate in every stage of research;Dan;Dev. Med. Child Neurol.,2023
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