Download our whitepaper,

Transforming Rett Syndrome
Research: Lessons from Successes
and Failures in Clinical Trial

What's Inside:

  • Key FDA-Approved Pivotal Endpoints for Rett Syndrome Clinical Trials.
  • Optimizing Recruitment with Adaptive Trial Designs for Rett Syndrome.
  • Comprehensive Analysis of FDA-Approved Rett Syndrome Drugs.
  • How Diagnostic Criteria for Rett Syndrome Have Evolved and Impact Research.
  • Overcoming Common Pitfalls in Rett Syndrome Clinical Trials.
  • And additional insights to drive your trial’s success.

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    Rett Syndrome

    Rett syndrome is a rare and severe, neurodevelopmental disorder primarily affecting females, caused by mutations in the MECP2 gene.

    It typically manifests after an initial period of normal development, with symptoms emerging between 6 to 18 months of age. These include severe loss of motor skills, communication abilities, and purposeful hand movements, often accompanied by repetitive hand stereotypies, such as hand-wringing or clapping.

    Cognitive impairments, breathing irregularities, and seizures are also common.

    iNGENū’s team of researchers and clinicians is dedicated to advancing Rett syndrome research. Through innovative trial designs and a patient-centered approach, we work to accelerate the development of new treatments that could offer improved options and outcomes for those affected by Rett syndrome.

    99%

    of cases arise due to random genetic mutations rather than being inherited

    50%

    of of individuals experience seizures, ranging from mild to severe

    1 in 5

    50%

    individuals have reduced pain sensitivity, which can delay recognition of injuries or illnesses

    Our clinical team has over

    120 years

    of combined clinical trial experience