New discoveries about the severe developmental disorder known as Rett syndrome could open the door to better treatments for the devastating, life-shortening condition. Scientists investigating the ...

Real World Data from LOTUS Study Evaluating Long-term Efficacy and Tolerability Outcomes of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Developmental Medicine and Child Neurology ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that Health Canada has granted marketing authorization of DAYBUE™ (trofinetide) for the treatment of Rett ...

Researchers have gained new insights into the molecular changes leading to Rett syndrome, a severe neurological disorder caused by mutations in the MeCP2 gene encoding methyl-CpG binding protein 2 ...

Canadian Rett Syndrome Coalition Demands Justified Reconsideration Under Time-Limited Access Framework TORONTO, Oct. 21, 2025 /CNW/ – The Canadian Rett Syndrome Coalition – comprising the ...

UAB will study a drug originally developed for Parkinson’s disease that may help reduce breath holding in patients with Rett syndrome. Pediatric neurologist Alan Percy, M.D., is a leading clinician ...

(Mass Appeal) - It's a rare and cruel disorder with symptoms that can begin as early as 6-months old--Rett Syndrome. Children with Rett Syndrome require round the clock care for life. Rett Syndrome ...

The Risers 4 Rett (R4R) women’s fly-fishing event and the Girls on the Rise fly-fishing clinic return to Minturn this weekend. Both events raise awareness for Rett Syndrome and support the work of the ...

NEW YORK--(BUSINESS WIRE)--Neurogene Inc. (Nasdaq: NGNE), a clinical-stage company founded to bring life-changing genetic medicines to patients and families affected by rare neurological diseases, ...

-- Findings reinforce efficacy and tolerability profile in patients prescribed DAYBUE in the U.S. for as long as 12 months, consistent with the Phase 3 LAVENDER™ trial that supported FDA approval and ...