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NON PROFIT

Rett Syndrome Research Trust campaign for Rett: There Is Hope

 Rett Syndrome Research Trust is on a mission to REVERSE Rett Syndrome.

Creative Activists

Linda Taylor

Jason Rem

What Is Rett Syndrome ?

Imagine the symptoms of autism, cerebral palsy, Parkinson’s, epilepsy, and anxiety disorders…all in one little girl.

Now imagine these symptoms vanishing … one by one.

This is the work of the Rett Syndrome Research Trust.

The Rett Syndrome Research Trust (RSRT) is the premier organization devoted exclusively to promoting international research on Rett Syndrome and related MECP2 disorders.

The goal is clear: to heal children and adults who will otherwise suffer from this disorder for the rest of their lives.

The story behind Rett Syndrome is complicated. It involves a devastating genetic affliction that starts with young girls and includes incredible family dynamics, groundbreaking treatment, care and science.

In a laboratory in Edinburgh University Adrian Bird, Ph.D and his associates have been proven that the symptoms of Rett Syndrome can be reversed in a mouse model. They are working on the identification of the brain regions involved with symptoms of the disease and are creating novel treatment approaches. This knowledge will lay the foundation for potential gene therapy or protein replacement approaches directed to specific brain regions of individuals with Rett Syndrome and MECP2 spectrum disorders including Autism.

The Rett developments of the last few years towards a cure have excited the scientific community. The hope is that these treatments are the mere tip of the iceberg.  Huda Zoghbi, M.D. examined  her first patient with Rett Syndrome  in the mid 1980’s and was so emotionally and intellectually hooked that she decided to put her nascent neurology clinical practice on hold and move instead into basic science. Her ambitious goal to locate the gene mutations responsible for this puzzling disorder was successfully realized sixteen years later.

Rett and MECP2 disorders are not neurodegenerative. The neurons in these girls’ brains are healthy and waiting to be restored.

  • Rett Syndrome is the most physically disabling of the autism spectrum disorders.

  • It strikes at random in early childhood, affecting little girls almost exclusively.

  • Many girls live into adulthood, requiring total, 24-hour-a-day care.

  • There is no treatment beyond supportive, and often ineffective, measures such as feeding tubes, bracing, orthopedic and GI surgeries, and medications for anxiety and seizures.

  • First recognized only 25 years ago, the prevalence of Rett Syndrome equals that of Cystic Fibrosis, Huntingtons and ALS but is vastly underfunded in comparison to those disorders

Symptoms include:

  • Loss of speech and motor control

  • Functional hand use replaced by compulsive hand movements

  • Disordered breathing

  • Severe digestive problems

  • Orthopedic abnormalities including scoliosis and fragile bones

  • Disrupted sleep patterns

  • Extreme anxiety

  • Seizures

  • Impaired cardiac and circulatory function

  • Parkinsonian tremor

How you can take action:


Support this project Rett: There Is Hope

Donate – All donations are tax deductible

Contact RSRT with your own idea of how to support this cause.

Attend RSRT events and fundraisers.

Volunteer to help with RSRT events and fundraisers.

Download the RSRT toolbar – It’s a safe and free way to support our research efforts.

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