Rett Syndrome Research Trust Appoints Amy Gilliland to its Board of Trustees
Amy’s daughter, Ashley, who was diagnosed with Rett syndrome in 2015, is the inspiration behind her drive to help find a cure ...
Marvel Announces First of Two Final Data Sets from Rett Syndrome Study: MB-204 Significantly Outperforms Trofinetide
Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF) and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively ...
PharmaTimes2d
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 ...
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.
PharmiWeb9d
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 (CNS) disorders, today announces that the first patient in the TREE study ...
Marvel Biosciences Corp.: Marvel Announces First of Two Final Data Sets from Rett Syndrome Study: MB-204 Significantly Outperforms Trofinetide
Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF) and its wholly owned subsidiary, Marvel Biotechnology Inc. (collectively the "Company" or "Marvel") ...
Daily Herald9h
Man gets 25 years for sexual assault on disabled woman
The man who sexually assaulted a profoundly disabled woman and impregnated her was sentenced Friday to 25 years in prison.
ProQR Announces Year End 2024 Operating and Financial Results
Axiomer™ ADAR-mediated RNA editing pipeline advancing across liver and CNS programs, with CTA filing on track for Q2 2025 for ...
Investing3d
Taysha Gene Therapies’ SWOT analysis: stock’s potential in Rett syndrome treatment
Preliminary data presented at the 2024 Rett Syndrome Scientific meeting demonstrated safety and consistent improvements across clinical domains for Rett syndrome symptoms. This early success has ...
Morningstar10d
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
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 ...