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.
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 ...
Investing10d
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 ...
Investing10d
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 ...
PharmaTimes10d
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 ...
Marvel Biosciences Corp.: 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. (collectively the "Company" or "Marvel") ...
Mena FN19d
Biomed Industries, Inc. To Announce Phase 2/3 Results Of NA-921 For Treating Rett Syndrome At The IRSF Conference 2025
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 ...
The Globe and Mail5d
Marvel to File for Orphan Drug Designation with The U.S. FDA for MB-204 as a Treatment for Rett Syndrome
Orphan Drug Designation is a critical regulatory pathway that supports the development of treatments for rare diseases affecting fewer than 200,000 individuals in the U.S. Rett Syndrome ...
Morningstar17d
Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med
in Patients with Rett Syndrome Published in Med -- Study supported FDA and Health Canada approvals of DAYBUE for treatment of Rett syndrome in patients ages two years and older -- Results ...