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 ……
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…
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…
Girls with Rett syndrome have significantly smaller brain volumes across all its regions, regardless of their age, a MRI study shows. Reductions in the size of certain brain regions were tied to the clinical severity of Rett patients, particularly in gross motor function, such as sitting and walking. Still,…
Bone mineral density, an indicator of bone strength, is significantly lower in girls and young women with Rett syndrome compared with healthy girls and women of the same age, a study shows. Reduced bone mineral density was tied to small body size, impaired mobility, and the use of the…
NLX-101 improves breathing and cognitive function in a female mouse model of Rett syndrome, a study found. The potential therapy activates the receptors that bind serotonin, a signaling molecule found at low levels in the brains of Rett patients. “This work provides compelling evidence of the therapeutic potential of…
One and a half years of daily Daybue (trofinetide) treatment safely improved communication, eye contact, and hand use among girls ages 2-4 with Rett syndrome, according to the final published results of the DAFFODIL trial, which tested the approved therapy in the youngest of patients. Although most patients…
Computer modeling confirms the weight-based dosing strategy for Daybue (trofinetide) to treat people with Rett syndrome, ages 2 and older. That’s according to two, back-to-back studies published separately in Advances in Therapy. Daybue, from Acadia Pharmaceuticals, is the first therapy approved in the U.S. for people with…
Defects in how cells degrade old or damaged components, a cellular recycling process called autophagy, contribute to the dysfunction of nerve cells in Rett syndrome, a lab study reports. Treating mice in a Rett disease model with trehalose, a naturally occurring molecule known to induce autophagy, restored the recycling…
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