Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and ...

LUCY Glenn appeared “completely healthy” until she was 18 months old when her behaviour suddenly changed. Her mum, Katherine McCready, 31, said noticed she suddenly stopped playing ...

Rett syndrome is a devastating neurological disorder ... such as epileptic seizures. In the phase 1/2 trial, the first four patients all achieved a "much improved" rating on the clinician-rated ...

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

Neurotech International Ltd. ( ($AU:NTI) ) just unveiled an update. Neurotech International Limited announced the publication of its Phase I/II ...

The first of which is that the company is gearing up to release additional results from its phase 1/2 study using NGN-401 for the treatment of women with Rett Syndrome. Such additional data is ...

Biomed Industries, Inc. to Announce Phase 2/3 Results of NA-921 for the treatment of Rett Syndrome at the IRSF Rett Syndrome Scientific conference 2025 The Phase 2/3 results of NA-921 shows strong ...

The long-term Phase 2/3 study demonstrated DAYBUE had a similar safety profile in children two to four years of age as that observed in individuals with Rett syndrome five years of age and older ...

The study, which included 15 patients, demonstrated that DAYBUE maintained a similar safety profile in younger pediatric patients as seen in older patients in previous Phase 3 studies. The ...

Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of leriglitazone in treating Rett syndrome. The trial involves 24 female patients ...