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

Dr. Budden is Associate Professor of Pediatrics at Oregon Health Sciences University in Portland, Ore., and Medical Director, Pediatric Development Program, Legacy Emanuel Children's Hospital ...

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

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, a late-stage biotech company focused on the development of therapies for orphan central nervous system ...

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 ...

Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that the journal Med published results from the open-label study, DAFFODIL™, evaluating the safety, tolerability and exploratory efficacy of ...

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 ...

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 ...

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 ...