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1 month ago | 
rettsyndromenews.com | Steve Bryson |Patricia Inacio |Jackie Babiarz |Marisa Wexler
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.
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1 month ago | 
rettsyndromenews.com | Marisa Wexler |Jackie Babiarz
Treatment with NA-921 was found to improve measures of abnormal behavior and overall health in girls and young women with Rett syndrome in a Phase 2/3 clinical trial. Data also suggested the oral therapy is less likely to cause digestive side effects than Daybue (trofinetide), which is marketed by Acadia Pharmaceuticals and currently the only approved treatment for Rett.
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2 months ago | 
rettsyndromenews.com | Marisa Wexler |Katherine Poinsatte |Jackie Babiarz |Patricia Inacio
A new study aims to raise awareness about Rett syndrome in boys and explore how the disorder affects them and their families. The project is being led by Talia Thompson, PhD, and Timothy Benke, MD, PhD, both professors at the University of Colorado. It’s funded by the International Rett Syndrome Foundation and Rocky Mountain Rett Association.
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2 months ago | 
rettsyndromenews.com | Marisa Wexler |Jackie Babiarz
Treatment with Daybue (trofinetide) in clinical trials helped girls and young women with Rett syndrome to communicate and use their hands better, according to interviews with caregivers of participants in two studies that tested the now-approved therapy.
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Jan 22, 2025 | 
rettsyndromenews.com | Esteban Cerezo |Ana Pena |Ana Peña |Jackie Babiarz |Patricia Inacio
Digestive problems, such as constipation and bloating, affect more than 80% of girls and women with Rett syndrome, a study reveals. The most frequent problems included constipation, difficulties with eating and swallowing, and gas and bloating. More than half of the patients showed irritability due to stomach or gut discomfort. Use of medications was associated with significantly better digestive health.
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Jan 8, 2025 | 
rettsyndromenews.com | Andrea Lobo |Steve Bryson |Marisa Wexler |Jackie Babiarz
About two in three girls with Rett syndrome develop scoliosis, a sideways curvature of the spine, while one in two exhibit foot deformities, according to a review study. About 30% of the patients experienced hip dislocations, the study found. In general, scoliosis was more prevalent in studies with a higher proportion of patients with MECP2 mutations, the main cause of Rett, and those enrolling patients with a mean age above 13.
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Dec 11, 2024 | 
rettsyndromenews.com | Steve Bryson |Jackie Babiarz |Patricia Inacio
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 process and improved nerve cell function, as well as the animals’ motor and exploratory skills. Its use also eased anxiety-like behaviors.
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Nov 20, 2024 | 
rettsyndromenews.com | Steve Bryson |Jackie Babiarz |Marisa Wexler
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 response associated with exposure to high doses of the adeno-associated virus (AAV), the gene therapy’s delivery vehicle.
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Nov 6, 2024 | 
rettsyndromenews.com | Margarida Maia |Andrea Lobo |Jackie Babiarz |Katherine Poinsatte
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.
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Oct 23, 2024 | 
rettsyndromenews.com | Marisa Wexler |Jackie Babiarz |Steve Bryson
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. The company said it will work to bring the newly approved treatment to patients as quickly as possible, but it didn’t give a specific timeline.
