WHAT IS RETT SYNDROME?

Imagine this: A young girl afflicted with several major disorders – Autism, Cerebral Palsy, Parkinsons, Epilepsy, and Anxiety.

Rett Syndrome is a genetic neurological disorder that almost exclusively affects girls. It strikes randomly, typically at the age of 6 to 18 months, and is caused by random mutations of the MECP2 gene on one of the X chromosomes.

Rett Syndrome deprives young children of speech, hand use, normal movement often including the ability to walk. The disorder brings anxiety, seizures, tremors, breathing difficulties, severe gastrointestinal issues and often Scoliosis – a curvature of the spine. While their bodies suffer, it is believed that their cognitive abilities remain largely intact. Although most children survive to adulthood, they require total round-the-clock care.

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RETT Founders' Story

1 in 10,000

girls are
affected with the disorder

Every 90

minutes a girl is
born with Rett Syndrome

350,000

girls and women
live with Rett Syndrome

ROADMAP FOR A CURE

The Rett Syndrome Research Trust’s 3-Year Strategic Plan to a Find Cure

Gene Therapy

A single gene therapy treatment
could be a one-time fix

MECP2 Reactivation

Wake up healthy copies of
the MECP2 gene

RNA Modifications

Potential modification of the
gene at the RNA level

Protein Replacement

Bypass the gene to provide the
needed proteins to cells

*Used with the permission of RSRT

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#ReverseRett

*Used with the permission of RSRT

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