top of page

Living with

Rett syndrome

Our precision therapeutic candidate platform is inspired by extraordinary people of all ages, cultures and backgrounds.

What is Rett syndrome?

Rett syndrome is a rare genetic neurological disorder that is first recognized in infancy and primarily occurs in girls, but there are rare cases reported in boys as well. Rett syndrome leads to severe impairments, affecting nearly every aspect of the person’s life. Finding trusted information that is reliable, current and vetted by global experts in the field of Rett syndrome is often a family’s first step after receiving a diagnosis.


Signs & Symptoms

The signs and symptoms of Rett syndrome are variable in severity and frequency and emerge at different times during the course of condition. After a relatively normal infancy, cognitive and motor delays begin to manifest along with slower head growth. Around 1.5 to 3 years of age, loss of spoken language and hand skills begins to manifest.

Loss of Expressive LanguageMonochrome.png

Loss of Expressive Language

Loss of Fine Motor SkillsMonochrome.png

Loss of Fine Motor Skills

Inability to WalkMonochrome.png

Impaired Ability to Walk

Rett syndrome has four core symptoms

Repetitive Hand MotionsMonochrome.png

Repetitive Hand Movement

Rett syndrome is associated with many more symptoms, including breathing and sleeping abnormalities, seizures, scoliosis, abnormal muscle tone and gastrointestinal disorders.


There are about 350,000 patients with Rett syndrome worldwide and about 11,000 patients in the United States. Rett syndrome is the second most common cause of severe intellectual disability in females and, during its period of developmental regression, a substantial proportion of affected individuals meet diagnostic criteria for autism spectrum disorder (ASD).

bottom of page