Experimental drug NLX-101 can relieve two of the most serious symptoms: irregular breathing and cognitive deficits.
A Rett síndrome It is a rare genetic neurological disease. Mainly affects women (only one in 10,000) and is caused in most cases by a MUTATION IN MECP2 – Located on the X chromosome.
Affected people usually have a normal development in the first months of life, but end up gradually lose capabilities already acquired as walking and speaking.
Many go through episodes of apnea, which have a strong impact on their quality of life and can aggravate other symptoms.
Rett Syndrome No cure. Although there are treatments that help control symptoms and improve the quality of life of patients.
In this context, a drug was tested in the University of Minho it can “Open doors” In this disease, as read in a statement sent to ZAP.
A team from the Institute of Research on Life and Health Sciences (ICVS) of the University of Minho School of Medicine indicates that it has taken an important step towards new therapies for Rett’s syndrome.
The experimental drug is called Nlx-101. Shows it that can relieve two of the most serious symptoms: a irregular breathing and the cognitive deficits.
In this analysis, early and continuing administration of the drug in a rodent that replicates various aspects of the disease prevented its cognitive deficits. In addition, after a single administration of the drug, the episodes of apnea were significantly reduced.
This drug was designed to act exclusively in a subtype of receivers of the serotonin neurotransmitter, the 5-HT1A, In specific brain zones linked to cognition and breathing control, allowing you to get the desired therapeutic effects and avoiding adverse effects.
Researcher Daniela Monteiro-Fernandes points out that NLX-101 “may contribute to the significant improvement of weakening symptoms of the disease, such as cognitive deficit or apnea, which often endanger the lives of patients”.
It is a study that paves the way for New Therapies directed, with the potential to transform care provided to those who live with Rett’s syndrome. But more studies need to be carried out to evaluate the safety and effectiveness of the drug in humans.
