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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…
Brain transplant of neural precursor cells, which are self-renewing cells that give rise to neurons and other cells in the brain, can reduce mortality, and restore memory and motor functions in a mouse model of Rett syndrome, according to a recent study. Genetic analysis revealed these cells may be…
During Rett Syndrome Awareness Month this October, the International Rett Syndrome Foundation (IRSF) invites the community to get involved and help raise awareness and funds for disease research and patient support. The foundation looks to expand on its $58-million investment in Rett research and support to date.
A surgical procedure called a corpus callosotomy, which severs the main connection between the two sides of the human brain, was found to reduce the seizure burden of a young girl with Rett syndrome and hard-to-treat seizures, according to a case report. Calling this a “unique case,” the…
Women and girls with Rett syndrome experience disease progression and increasing disease burden even as they rely on supportive therapies, a study found. Close to half of all patients in the study needed a hospital or emergency room visit, the researchers said. Children and adolescents saw “increasing disease severity…
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