Researchers at the Josep Carreras Leukaemia Research Institute revealed a new aspect of Rett Syndrome’s pathogenesis that can lead to new therapeutical approaches in the future. According to the ...

NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.

The patient affected by a serious adverse event in Neurogene's clinical trial of Rett syndrome gene therapy has now died, but that won't cause the study to be halted. The FDA has allowed the ...

The Blue Bird Circle Rett Center at Baylor College of Medicine is composed of a multidisciplinary team of health professionals. Several BCM physicians are involved clinically at the Blue Bird Circle ...

Rett syndrome affects 1 in 10,000 to 15,000 female ... whether Stoke's platform is a one-drug success story or a scalable mechanism that can be adapted to multiple indications.

DAYBUE, a synthetic analog of insulin-like growth factor 1, is designed to treat Rett syndrome, although its exact therapeutic mechanism is not fully understood. It is available as an oral ...

Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med -- Study supported FDA and Health Canada ...