Trofinetide in Rett syndrome: A brief review of safety and efficacy-Reference-Cited by-同舟云学术

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Trofinetide in Rett syndrome: A brief review of safety and efficacy

Published:2023-11-30 Issue:4 Volume:12 Page:262-266
ISSN:2186-361X
Container-title:Intractable & Rare Diseases Research
language:en
Short-container-title:IRDR

Author:

Singh Alok¹,Balasundaram Mahesh Kumar²,Gupta Dhyuti³

Affiliation:

1. Department of Pharmacology, All India Institute of Medical Sciences Raipur, Chhattisgarh, India.

2. Department of Pharmacology, Kasturba Medical College, Mangalore, Manipal Academy of Higher Education, Manipal, India.

3. Department of Pharmacology, Teerthanker Mahaveer Medical College and Research Centre, Moradabad, Uttar Pradesh, India.

Publisher

International Research and Cooperation Association for Bio & Socio-Sciences Advancement (IRCA-BSSA)

Subject

General Medicine

Link

https://www.jstage.jst.go.jp/article/irdr/12/4/12_2023.01060/_pdf

Reference20 articles.

1. 1. Good KV, Vincent JB, Ausió J. MeCP2: The genetic driver of Rett syndrome epigenetics. Front Genet. 2021; 12:620859.

2. 2. Hagberg B, Aicardi J, Dias K, Ramos O. A progressive syndrome of autism, dementia, ataxia, and loss of purposeful hand use in girls: Rett's syndrome: report of 35 cases. Ann Neurol. 1983; 14:471-479.

3. 3. Mount RH, Charman T, Hastings RP, Reilly S, Cass H. The Rett Syndrome Behaviour Questionnaire (RSBQ): refining the behavioural phenotype of Rett syndrome. J Child Psychol Psychiatry. 2002; 43:1099-1110.

4. 4. Operto FF, Mazza R, Pastorino GMG, Verrotti A, Coppola G.Epilepsy and genetic in Rett syndrome: A review. Brain Behav. 2019; 9:e01250.

5. 5. Tarquinio DC, Hou W, Berg A, Kaufmann WE, Lane JB, Skinner SA, Motil KJ, Neul JL, Percy AK, Glaze DG. Longitudinal course of epilepsy in Rett syndrome and related disorders. Brain. 2017; 140:306-318.

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1. Rett syndrome – advances in gene and trofinetide therapy;Aktualności Neurologiczne;2024-08-28

2. Is trofinetide a future treatment for Rett syndrome? A comprehensive systematic review and meta-analysis of randomized controlled trials;BMC Medicine;2024-07-18

3. Manufacturing Uniform Cerebral Organoids for Neurological Disease Modeling and Drug Evaluation;2024

4. Trofinetide approved for children and adults with Rett syndrome (RTT): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG);Genetics in Medicine Open;2024

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