Neurolixis has been awarded a new research grant from the Rett Syndrome Research Trust (RSRT) to advance development of NLX-101, a clinical-phase candidate for treatment of Rett syndrome. NLX-101 has previously been awarded Orphan Drug designation for this indication in both the USA and in the European Union.
Mutations in the methyl CpG binding protein 2 gene (MECP2) are the cause of the devastating childhood neurological disorder Rett Syndrome. Despite intense efforts spanning several decades the precise function of MECP2 has been difficult to pin down.
Researchers have discovered that the protein product of the gene MECP2, which is mutated in about 95% of Rett syndrome is a global activator of neuronal gene expression. Mutations in the protein can cause decreased gene transcription, reduced protein synthesis, and severe defects in the AKT/mTOR signaling pathway.
Dr Julian Paton's, University of Bristol, a preclinical breathing study into NLX-101 from Neurolixis, which is being part funded by Cure Rett through Rettsyndrome.org. Dr Paton's study tackles an often overlooked area of Rett ...
A team at Cold Spring Harbor Laboratory (CSHL) has developed a strikingly new approach for treating Rett syndrome, a devastating autism spectrum disorder (ASD) that affects 1 in 10,000 people in the US, mostly girls.
Bone marrow transplant does not rescue mouse models of Rett syndrome, a severe neurological disease, a new study shows. The findings contradict seemingly promising presults previously published by another laboratory.
Trumbull, CT (PRWEB) February 03, 2015 -- Today the Rett Syndrome Research Trust (RSRT) announced research investments of $5.8 million awarded in 2014. RSRT’s sole and urgent goal is to abolish Rett Syndrome and related disorders.
Drugs that activate the silent copy of the X chromosome in women may be able to undo the damage from mutations in genes located there. The study offers hope for treating Rett syndrome and other disorders linked to the X chromosome.
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