Mef2c Gene News-Rett and MEF2C Duplication Syndrome, MEF2C Deficiency etc.

24/09/2023

This may be good news.

Base editing, which makes specific changes to a cell’s genome, is put to the test in CAR-T-cell treatments for leukaemia.

13/03/2023

Article Extract:
"Trofinetide will treat Rett syndrome...Trofinetide, to be marketed as Daybue, is the first drug treatment for the condition."

A molecule discovered by Distinguished Professor Dame Margaret Brimble of the University of Auckland is the basis for a breakthrough neurological drug approved by the US Food and Drug Administration on March 10. Trofinetide will treat Rett syndrome, a ...

24/09/2022

https://www.streetinsider.com/Press+Releases/Neurogenes+Gene+Replacement+Program+is+the+Eighth+Biopharmaceutical+Company+Program+Facilitated+by+the+Rett+Syndrome+Research+Trust/20102556.html

TRUMBULL, Conn. (PRWEB) May 19, 2022 The research and resource development efforts of the Rett Syndrome Research Trust (RSRT) have helped generate an eighth biopharmaceutical program that tackles the root cause of Rett syndrome. Neurogene,...

23/09/2022

https://rettsyndromenews.com/news/non-toxic-gene-therapy-rett-syndrome-extends-lifespan-mice/

NGN-401, a new gene therapy in the pipeline at Neurogene, utilizes EXACT technology to avoid conventional gene replacement safety issues.

22/09/2022

https://www.pnas.org/doi/full/10.1073/pnas.1311685111

21/09/2022

https://reverserett.org/news/tags/mecp2-protein/

Rett Syndrome Research Trust advances medical research, news and information about Rett Syndrome. A singular guide for parents and the scientific and medical community.

20/09/2022

https://rettsyndromenews.com/news/rett-patients-have-fewer-monthly-seizures-cannabidivarin-treatment/

19/09/2022

https://www.cell.com/neuron/fulltext/S0896-6273(06)00010-9?_returnURL=https%3A%2F%2Flinkinghub.elsevier.com%2Fretrieve%2Fpii%2FS0896627306000109%3Fshowall%3Dtrue

18/09/2022

https://world.einnews.com/pr_news/590469784/acadia-pharmaceuticals-announces-trofinetide-new-drug-application-for-the-treatment-of-rett-syndrome-has-been-accepted-for-filing-and-review-by-u-s

18/09/2022

https://www.hcplive.com/view/rett-syndrome-treatment-receives-priority-review-from-fda

18/09/2022

https://www.consultantlive.com/view/rett-syndrome-treatment-receives-priority-review-from-fda

Acadia Pharmaceuticals announced that the FDA granted a priority review for trofinetide and set March 12, 2023 as the PDUFA date.

16/09/2022

https://etheses.whiterose.ac.uk/30808/

15/09/2022

https://rettsyndromenews.com/news/2022-kavli-prize-neuroscience-awarded-rett-gene-discoverer/

Neurologist Huda Zoghbi, MD, was awarded the 2022 Kavli Prize for her discovery of the MECP2 gene that causes most Rett syndrome cases.

14/09/2022

https://www.pedneur.com/article/S0887-8994(22)00105-9/fulltext

13/09/2022

https://freemedart.ru/optimized-administration-of-the-m4-pam-vu0467154-demonstrates-broad-efficacy-but-limited-effective-concentrations-in-mecp2-mice-by-jakub-cikowski/

12/09/2022

https://onlinelibrary.wiley.com/doi/10.1002/hipo.23455

12/09/2022

https://rettsyndromenews.com/news/elevated-mir-101a-underlie-processes-leading-symptoms-mice/

Levels of a small RNA molecule called miR-101a may be elevated in certain brain regions of Rett syndrome patients, a preclinical study says.

12/09/2022

https://pubmed.ncbi.nlm.nih.gov/36082308/

10/09/2022

http://www.informatics.jax.org/allele/genoview/MGI:5491051?counter=20

View Mecp2/Y involves: 129P2/OlaHsd * FVB: phenotypes, images, diseases, and references.

09/09/2022

https://newsfounded.com/malaysia/mecp2-protein-treatment-extends-the-life-of-rett-syndrome-mice/

MeCP2 Protein Treatment Extends the Life of Rett Syndrome Mice Health 0 Views Share Facebook Twitter LinkedIn Pinterest Treatment with a modified version of the MeCP2 protein, one designed to better pe*****te cells, dramatically extended lifespan in a mouse model of Rett syndrome, a study reports. T...

09/09/2022

https://rettsyndromenews.com/news/treatment-mecp2-protein-extends-lifespan-rett-sydrome-mice/

Treatment with a version of the MeCP2 protein was found to markedly extended lifespans in a mouse model of Rett syndrome, a study reports.

08/09/2022

https://pubmed.ncbi.nlm.nih.gov/35702943/

Our study is one of the largest cohorts conducted on MDS individuals characterizing the frequency and severity of MDS symptoms. Additionally, these study results will contribute to establishing a foundation to develop parent-reported outcomes in MDS.

07/09/2022

https://paperity.org/p/81014105/d-cycloserine-improves-synaptic-transmission-in-an-animal-mode-of-rett-syndrome

Paperity: the 1st multidisciplinary aggregator of Open Access journals & papers. Free fulltext PDF articles from hundreds of disciplines, all in one place

06/09/2022

https://reverserett.org/news/tags/mecp2/

Rett Syndrome Research Trust advances medical research, news and information about Rett Syndrome. A singular guide for parents and the scientific and medical community.

05/09/2022

https://www.sciencedirect.com/science/article/abs/pii/S0387760404001962

About 80% of female patients with Rett syndrome (RTT) display a mutation in the methyl-CpG-binding protein 2 (MECP2) gene, but most males with MECP2 m…

05/09/2022

https://journals.sagepub.com/doi/abs/10.1177/08830738030180100901

The association of Rett syndrome with pathogenic mutations of the methyl-CpG binding protein 2 ( MECP2) gene was first made in 1999. Since that time, it has bee...

03/09/2022

https://psychiatry.dev/tts/2022/06/disruption-of-mecp2-tcf20-complex-underlies-distinct-neurodevelopmental-disorders-pubmed-2/

02/09/2022

https://www.technologynetworks.com/neuroscience/news/brain-organoids-show-reveal-link-between-muscular-dystrophy-and-rett-syndrome-363174

01/09/2022

https://seekingalpha.com/news/3857763-acadia-pharma-submits-marketing-application-to-fda-for-trofinetide-to-treat-rett-syndrome

Acadia Pharma (ACAD) said on Monday it had submitted its marketing application to the U.S