NGN-401, Neurogene‘s experimental gene therapy for Rett syndrome, was designated a regenerative medicine advanced therapy (RMAT) by the U.S. Food and Drug Administration (FDA). Based on preliminary data from an ongoing Phase 1/2 trial (NCT05898620), which is testing the gene therapy in girls with Rett, the…
The U.S. Food and Drug Administration (FDA) has selected Neurogene‘s NGN-401, an experimental gene therapy for Rett Syndrome, for its Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. As part of START, which the FDA launched in September 2023, Neurogene will have enhanced communications with…
MeCP2, the protein impaired in most cases of Rett syndrome, controls the production of alpha-synuclein, a protein implicated in Parkinson’s disease, a study reports. Alpha-synuclein production was highest when the MeCP2 protein carried mutations from Rett patients with symptoms similar to those of Parkinson’s, data showed. “MeCP2 is…
The Rett Syndrome Research Trust (RSRT) has partnered with Vivalink to use its Multi-Vital ECG wearable patch to monitor heart rate in people with Rett syndrome participating in the observational VIBRANT study. VIBRANT (NCT06338267) is set to enroll about 30 Rett patients at sites…
New research supports how the lack of Mecp2 protein impairs the function of healthy nerve cells in a mouse study of Rett syndrome. Specifically, the function of nerve cells was affected when the cells were grown alongside nerve-cell supporting astrocytes lacking the Mecp2 protein, mimicking the genetic defect that…
A lack of MECP2 protein, the chief underlying cause of Rett syndrome, prevented precursor cells from transforming into fully mature nerve cells in the early stages of brain development, a study suggests. Using 3D organoids that mimic the brain, blocking a pathway elevated by MECP2 depletion enhanced the formation…
The International Rett Syndrome Foundation (IRSF) has launched the My Rett Ally app as a digital tool to help parents and caregivers of people with Rett syndrome, calling it “a valuable addition to the resource toolkit.” Free to those who care for Rett patients in the U.S., the…
The first patient has been dosed with TSHA-102, an investigational gene therapy by Taysha Gene Therapies, to treat Rett syndrome. The Phase 1/2 trial, dubbed REVEAL (NCT05606614), is still recruiting up to 12 women with Rett syndrome at its single site in Montreal, Canada. Early safety data…
Daybue (trofinetide) is easing the neurobehavioral symptoms of Rett syndrome with acceptable safety in girls ages 2 to 4, according to early data from the open-label DAFFODIL Phase 2/3 trial. These results were consistent with previous clinical trials in Rett patients starting at age 5 that supported…
Mice in Rett syndrome models showed a loss of brain volume before disease symptoms were evident, with brain shrinkage worsening over time, an MRI study showed. Delays in brain growth were observed in these mice, and brain growth stopped and appeared to reverse in older female Rett mice. Because…
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