“Lucy is such an infectious, happy, loving person and I’m so proud and so grateful for her,” Katherine said. “We know if she ...

This means the other half of the cells with a wild-type copy of MECP2 inherited from the other parent expresses normal MeCP2 ...

Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...

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

Amy’s daughter, Ashley, who was diagnosed with Rett syndrome in 2015, is the inspiration behind her drive to help find a cure ...

Neurotech said Rett syndrome was a rare genetic neurological and developmental disorder and is almost exclusively the result of mutation(s) in the methyl CpG binding protein 2 (MECP2) gene located on ...

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 ...

Neurogene has reported encouraging efficacy results with its gene therapy for rare genetic disorder Rett syndrome, but investors' concerns about safety seem to be weighing on its share price.

Phase 2a TREE study assesses safety and efficacy of leriglitazone Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of leriglitazone ...

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 ...

which is required for normal brain development and function. Rett syndrome occurs almost exclusively in girls, with an incidence of one in 10,000 female live births. The prevalence is ~15,000 ...