Rett Syndrome
Rett Syndrome is a rare and complex neurological disorder that profoundly impacts nearly every aspect of an individual’s life. It is characterized by severe developmental regression, loss of speech, motor impairments, intellectual disabilities, and often, breathing irregularities. Individuals with Rett Syndrome may also experience seizures, repetitive hand movements, and other serious health challenges. Without appropriate care and intervention, the condition significantly affects independence and quality of life.
Advances in genetic research have deepened our understanding of Rett Syndrome, particularly its connection to mutations in the MECP2 gene. This knowledge has opened the door to more targeted therapeutic approaches aimed at addressing the underlying genetic and molecular mechanisms of the disorder. Emerging therapies hold promise for improving neurological function and enhancing developmental outcomes, offering hope for individuals and families navigating this condition.
At iNGENū, we are committed to advancing research and innovation for Rett Syndrome. Through cutting-edge science and a patient-centered focus, our team of researchers and clinicians strives to accelerate the development of effective treatments, aiming to improve the quality of life and bring hope to those impacted by this rare and challenging disorder.
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
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
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In light of the impact Rett Syndrome has on individuals and society as a whole, continued research efforts are crucial to improve diagnosis, treatment, and ultimately, patients' quality of life.
The development of clinical trial protocols that meet FDA approval standards is crucial for the success of new Rett Syndrome treatments.