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 Guardian6d
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, ...
PharmaTimes12d
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 ...
Investing12d
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 ...
UNILAD3d
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' ...
Business Insider11d
Rett Syndrome Research Trust Appoints Amy Gilliland to its Board of Trustees
Multiple clinical trials in gene therapy for Rett syndrome are now underway, all possible because of RSRT-funded research. In 2024 RSRT launched a $40 million initiative, Roadmap to Cures, with a goal ...
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 ...
Investing12d
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 ...