Acadia Pharmaceuticals’ DAYBU Approved in EU for Rett Syndrome
SAN DIEGO, U.S, Aug 28, 2026 Acadia Pharmaceuticals Inc. announced that the European Commission (EC) has granted marketing authorization...
Rett syndrome
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Minoryx Therapeutics Secures CHMP Backing for NEZGLYAL® in cALD
Barcelona, Spain and Düsseldorf, Germany – July 24, 2026 Minoryx Therapeutics and Neuraxpharm Group have received a positive opinion...
Acadia’s DAYBU Recommended for EU Approval in Rett Syndrome
SAN DIEGO, June 26, 2026 Acadia Pharmaceuticals announced that the Committee for Medicinal Products for Human Use (CHMP) of...
Taysha Gene Therapies Showcases TSHA-102 Progress at 2026 IRSF Meeting
DALLAS, June 25, 2026 Taysha Gene Therapies announced multiple presentations highlighting the clinical progress of its investigational TSHA-102 gene...
JW Pharmaceutical Showcases DDC-02 Potential Across Multiple Rare Neurodevelopmental Disorders at WODC USA 2026
BOSTON, Mass., June 11, 2026 JW Pharmaceutical announced new preclinical findings and its global development strategy for DDC-02, an...
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Neurogene Highlights Rett Gene Therapy Progress at ASGCT
NEW YORK, May 12, 2026 Neurogene presented new data at the 2026 American Society of Gene and Cell Therapy...
Acadia Expands DAYBUE STIX Access for Rett Syndrome
San Diego, California, USA, April 7, 2026 Broad U.S. Availability Expands Patient Access In a major advancement for rare...
Neurogene Shows Strong Pediatric Gains in NGN-401 Rett Trial
NEW YORK—Nov. 12, 2025—Neurogene Inc. reported positive interim data from the pediatric cohort of its Phase 1/2 trial evaluating...
FDA Breakthrough Status Advances Rett Syndrome Gene Therapy
DALLAS, Oct. 2, 2025 — The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to TSHA-102,...
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