Blocking a small RNA molecule that helps control whether certain genes are switched on or off reactivated the healthy copy of the MECP2 gene in a mouse model of Rett syndrome, increasing lifespan and activity, and improving breathing. While blocking miR‑106a appears to be “a feasible therapeutic strategy,” before…
Vorinostat, an approved lymphoma treatment sold as Zolinza, was found to ease both neurological and non-neurological symptoms in a mouse model of Rett syndrome in a new study. When an oral formulation of vorinostat was administered after symptom onset, the drug was even more effective than Daybue (trofinetide),…
Cannabidivarin (CBDV), a non-psychoactive compound found in cannabis, preserves cognitive function and improves motor coordination in a female mouse model of Rett syndrome, a study suggests. “These findings provide new insights into the role of CBDV in [Rett] and support for future research, highlighting its potential as a ……
NTI164, a cannabis-based oil being developed by Neurotech International, may help girls with Rett syndrome communicate more easily, enjoy better moods, and have a better quality of life with few side effects, a small study in Australia found. The study, “Full-Spectrum Medicinal Cannabis Plant Extract 0.08% THC (NTI164)…
“The Clinical Severity Scale (CSS) remains a relevant tool in assessing disease severity in Rett syndrome as it reflects core clinical criteria and mirrors concerns from parents or caregivers, but its role in gauging outcomes is limited, a study suggests.” CSS “continues to be…
A lack of awareness or misconceptions in the medical community about Rett syndrome occurring in boys and men commonly leads to misdiagnoses or diagnostic delays, according to a new study. “Diagnostic providers must increase their awareness of males with [Rett] and be prepared to deliver the diagnosis with empathy…
Many types of movement disorders such as uncontrolled muscle contractions and Parkinson’s-like motor changes often co-occur with Rett syndrome, a study shows. The severity of movement disorders is associated with Rett’s overall severity, with uncontrolled muscle contractions particularly linked with breathing trouble and digestive problems, data indicate. The findings…
Neurogene has received written agreement from the U.S. Food and Drug Administration (FDA) for a registrational trial of NGN-401, a gene therapy candidate for Rett syndrome. A registrational, or pivotal, trial is designed to provide the evidence needed to support an application for FDA approval. “We appreciate the…
An aspiration event— when something other than air enters the lungs — increases the risk of lower respiratory tract infection and respiratory failure in people with Rett syndrome, a study found. Patients with known or suspected aspiration events more commonly had risk factors like epilepsy and difficulty swallowing, and…
Changes in auditory event-related potentials, or AEPs, the brain’s electrical activity in response to sound, may serve as a non-invasive biomarker to assess treatment response in people with Rett syndrome, a study suggested. Depressed AEPs, as measured by electroencephalography (EEG), which detects the brain’s electrical activity, were normalized when…
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