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 ...

Rett syndrome affects one in every 12,500 female births ... Percy hopes that one day a treatment will be devised that can reverse the developmental regression of Rett patients. Stem-cell and ...

Inspired by her daughter Adalyn who lives with Rett Syndrome, Smith is hosting inclusive mini-photo ... This condition can ...

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

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

Long-term clinical profiles of female patients with classic Rett syndrome are presented and exemplified by three cases, as experienced over four decades. Emphasized is the frequently surprisingly ...

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 ...

“I took the call, and when they told me it was Rett syndrome, I remember it sounding familiar. I said, ‘Don’t tell me this is the one where the child loses skills, where it causes regression?’ ...

Eventually her daughter was diagnosed with Rett Syndrome, a rare genetic neurological ... the condition is usually undetected until a major regression occurs at around two years of age, when ...

"The recently announced interim data from participants dosed with 1E15 vg in the NGN-401 clinical trial showed consistent improvements across multiple domains that define Rett syndrome ... the phase ...

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 ...

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 ...