Neurogene: Potential To Target Root Cause Of Rett Syndrome With NGN-401
NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.
PharmaTimes4d
Minoryx doses first patient with leriglitazone in Rett syndrome study
Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of ...
pharmaphorum11d
Neurogene shares fall on Rett syndrome gene therapy data
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 ...
AZoLifeSciences on MSN10d
Minoryx Announces Dosing of the First Patient with Leriglitazone in the Phase 2a Study in Rett Syndrome (TREE Study)
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 Treating Rett Syndrome At The IRSF Conference 2025
Biomed Industries, Inc. Biomed pipeline Biomed Industries, Inc. to Announce Phase 2/3 Results of NA-921 for the Treatment of Rett Sy ...
Morningstar12d
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
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 ...
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Acadia Pharmaceuticals Announces Positive DAFFODIL Study Data For DAYBUE In Rett Syndrome
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 ...
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
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 ...
PharmaTimes5d
Minoryx doses first patient with leriglitazone in Rett syndrome study
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 ...