Home Visit Nurses Deliver Complete Trial Data Despite A Challenging Population, A Pandemic And Severe Weather

Background

Rett syndrome, a rare progressively degenerative neurological disorder that occurs mostly in girls, is caused by mutations in the X-linked gene methyl-CpG-binding protein 2 (MECP2), a pervasive transcriptional regulator. At a very young age—often between 6 and 18 months of age—girls with classic Rett syndrome start developing severe problems with language and communication, learning, coordination and other brain functions, often accompanied by seizures. Rett syndrome is estimated to affect 6,000 – 9,000 patients in the U.S. alone.