“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 ...

Richard Engel and his wife Mary are coping with their 2-year-old son Henry's devastating diagnosis with Rett Syndrome Charlotte Triggs is a digital leader and veteran journalist with 20 years ...

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 ...

Its symptoms typically become apparent between the ages of 12 to 18 months. Rett syndrome is devastating as it deprives young children of speech, hand use, and normal movement often including the ...

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 ...

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 ...

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 ...