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 ...

SAN DIEGO--(BUSINESS WIRE)--Acadia Pharmaceuticals Inc. (Nasdaq: ACAD) today announced that four posters in Rett syndrome will be presented at the upcoming American Academy of Neurology (AAN) annual ...

Rett syndrome is a neurodevelopmental disorder that primarily affects girls, most of whom have mutations in the transcription regulatory gene MECP2. However, mutations in MECP2 also have been ...

Taysha Gene Therapies has unfurled more data in the developmental disorder Rett syndrome, linking its gene therapy to improvements in the first adults and children to receive low doses of the ...

Rett syndrome is a rare devastating neurological disorder that primarily affects young girls and manifests as an impaired ability to walk and talk, along with characteristic 'hand-wringing' movements, ...

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 ...

Partnership leverages Profluent’s frontier AI models to take on one of genetic medicine’s hardest problems: designing personalized genomic interventions for rare disease patients. Profluent Bio ...

Neurogene initiated an adolescent/adult Cohort 3 to gain initial data on the potential of NGN-401 to treat a broader patient population. The company also said it does not expect to move forward with ...

Credit: Getty Images. TSHA-102 is an investigational adeno-associated virus 9 gene transfer therapy. The Food and Drug Administration (FDA) has granted Fast Track designation to TSHA-102 for the ...

The legacy of Henry Engel, the 6-year-old son of NBC News' chief foreign correspondent Richard Engel, is still living on almost eight months after his death from Rett syndrome. Doctors who worked with ...

Accomplished nonprofit and biotech leader brings decades of rare disease experience to guide IRSF's next chapter of impact and growth. [Laura's] unique blend of scientific insight, policy experience, ...