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.
UNILAD1d
Girl, 13, 'locked' inside own body by incurable condition diagnosed after she did one thing as a child
A mom has opened up about her daughter developing 'completely normally until one day she woke up and something was different' ...
Marvel to File for Orphan Drug Designation with The U.S. FDA for MB-204 as a Treatment for Rett Syndrome
Marvel Biosciences Corp. (TSXV: MRVL) (OTCQB: MBCOF), and its wholly owned subsidiary, Marvel Biotechnology Inc.
Scioto Valley Guardian4d
Cincinnati researchers receive $2 million in grants for groundbreaking Rett Syndrome research
The International Rett Syndrome Foundation (IRSF), headquartered in Cincinnati, has announced the allocation of nearly $2 million in grants to seven leading researchers, ...
Investing9d
Taysha Gene Therapies’ SWOT analysis: stock’s potential in Rett syndrome treatment
The company has secured Regenerative Medicine Advanced Therapy (RMAT) designation for Rett syndrome, which could potentially expedite the approval process. An upcoming Type B meeting under the ...
PharmaTimes9d
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 ...
Neuren Pharmaceuticals: Ground-breaking impacts on neurodevelopmental disorders
Neuren Pharmaceuticals (ASX:NEU) is focused on neurodevelopmental disorders and rare disease drug development.In this ...
Rett Syndrome Research Trust Appoints Amy Gilliland to its Board of Trustees
I’m thrilled to join the Board of Trustees and get even closer to the Reverse Rett mission,” says Amy Gilliland. “As a parent of a child living with Rett syndrome—like so many others leading and ...
Investing9d
Taysha Gene Therapies’ SWOT analysis: stock’s potential in Rett syndrome treatment
The company has secured Regenerative Medicine Advanced Therapy (RMAT) designation for Rett syndrome, which could potentially expedite the approval process. An upcoming Type B meeting under the RMAT ...