Industry News Archives | International Rett Syndrome Foundation

March 31, 2025

European Commission grants NTI64 Orphan Drug Designation for Rett Syndrome

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Acadia

Acadia Pharmaceuticals Submits New Drug Application to the U.S. FDA for Trofinetide for the Treatment of Rett Syndrome

Alcyone

Alcyone Presents Preclinical Data on Gene Therapy Programs with The Center for Gene Therapy at The Abigail Wexner Research Institute (AWRI) at ASGCT 2022

Taysha

Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-102 in Rett Syndrome

Stoke Acadia

Acadia Pharmaceuticals and Stoke Therapeutics Announce Collaboration to Pursue Multiple RNA-based Treatments for Severe and Rare Genetic Neurodevelopmental Diseases

Acadia

Acadia Pharmaceuticals Announces Positive Top-line Results from the Pivotal Phase 3 Lavender Trial of Trofinetide in Rett Syndrome

Anavex

Anavex Life Sciences Announces ANAVEX®2-73 Meets Primary and Secondary Endpoints in Clinical Trial

Anavex

Anavex Life Sciences Announces First Patient Dosed in Phase 2 Clinical Trial of ANAVEX®2-73

AMO

AMO Pharma Receives FDA Orphan Drug Designation for AMO-04 in Treatment of Rett Syndrome

Neurolixis

European Commission approves Orphan Medicinal Product designation for NLX-101