Rett syndrome has a characteristic temporal profile that differs from that seen in neurodegenerative disorders. Following the period of developmental stagnation and then regression, stabilization ...

MECP2 mutations are the major genetic mutation associated with Rett syndrome. Diagnosis is made via observable features, which include regression of developmental features, including language and ...

Scientists investigating the severe developmental disorder known as Rett syndrome have discovered a series of crucial molecular changes that occur long before symptoms appear.

Researchers at Baylor College of Medicine, Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas Children’s Hospital and collaborating institutions have gained new insights into the ...

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

A patient has died after receiving the high dose of Neurogene’s Rett syndrome gene therapy candidate. The biotech had disclosed the adverse event Nov. 11, and explained the patient was in ...

ProQR Therapeutics NV. (Nasdaq: PRQR) (ProQR), a company dedicated to changing lives through transformative RNA therapies based on its proprietary Axiomer™ RNA editing technology platform, today ...

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 ...

Rett Syndrome, a rare and devastating neurodevelopmental ... followed by a period of developmental stagnation, then a regression in language and motor skills, followed by long-term relative ...