News

Many ways to show support for Rett Syndrome Awareness Month

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.

Rett patients see disease progression despite therapy: Study

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…

FDA gives NGN-401, potential Rett gene therapy, RMAT status

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…