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A new study sheds light on how MeCP2 protein dysfunction leads to brain abnormalities in Rett syndrome, and provides a framework to develop new treatments like gene therapies. Specifically, researchers conducted a study in adult mice and found the loss of the MeCP2 protein — not problems during development…
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…
A research team has identified Rett syndrome-specific molecules that regulate gene activity, called microRNAs, that are associated with the growth of patient-derived brain cells and may serve as biomarkers for monitoring Rett progression. Using 3D organoids that mimic the brain, the team identified these microRNA fingerprints within extracellular vesicles…
Marvel Biosciences‘ MB204, an experimental treatment for Rett syndrome, was shown to improve social behaviors — with “durable” effects — in a mouse model of the disease. Based on these results, Marvel intends to seek orphan and/or rare disease designation from the U.S. Food and Drug Administration (FDA)…
A girl with Rett syndrome participating in a clinical trial testing gene therapy NGN-401 experienced a serious, treatment-related immune reaction. The patient, who received a high dose of NGN-401 in an ongoing Phase 1/2 trial (NCT05898620), experienced signs of systemic (body-wide) hyperinflammatory syndrome, a rare and life-threatening immune…
The first four girls with Rett syndrome treated with a low dose of Neurogene‘s gene therapy candidate NGN-401 in a clinical trial have experienced new developmental milestones and seen meaningful gains in skills, according to new interim data. Clinicians and caregivers both have confirmed the improvements seen in…
Into-the-nose delivery of a solution containing clinical-grade human nerve growth factor (NGF) improved cognitive and motor function in a mouse model of Rett syndrome, including in male mice with more severe and rapidly advancing symptoms, a study has found. This could be “a non-invasive and effective route of administration…
A female mouse model of Rett syndrome manifested gene activity changes that began before the symptoms started and that mimicked those found in people with the condition. Biological pathways affected across the disease’s progression also overlapped with other neurodegenerative and addiction disorders. “Since RTT [Rett] in humans almost…
Health Canada has approved Daybue (trofinetide) for the treatment of Rett syndrome in adults and children ages 2 and older who weigh at least 9 kg (about 19.8 lbs). The decision makes Daybue the first approved Rett treatment in Canada, according to its developer Acadia Pharmaceuticals.
Increased levels of CB1 cannabinoid protein receptors, known as CB1Rs, may be tied to dysfunction of the brain’s mitochondria — its cellular energy-producing compartments — and to cognitive deficits in Rett syndrome, according to a study in a mouse model. The study also reported that blocking CB1Rs in the…
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