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

08/11/2024

https://www.dovepress.com/profile-of-trofinetide-in-the-treatment-of-rett-syndrome-design-develo-peer-reviewed-fulltext-article-DDDT

Profiling the use of Trofinetide in the treatment of Rett Syndrome: Investigating its design, development and potential place in therapy

20/02/2024

https://www.livescience.com/health/genetics/crispr-will-provide-cures-for-genetic-diseases-that-were-incurable-before-says-renowned-biochemist-virginijus-siksnys

Live Science spoke with biochemist Virginijus Šikšnys, whose work helped establish CRISPR as a gene-editing system.

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