Rett Syndrome and Fragile X are challenging neurodevelopmental disorders, each with unique manifestations. Rett Syndrome, primarily affecting girls, leads to severe impairments in motor skills and language, whereas Fragile X, more common in boys, is associated with intellectual disabilities, behavioral challenges, and distinct physical traits.
While current treatments mainly focus on symptom management, there is a significant need for therapeutic approaches that address the underlying genetic and neurobiological dysfunctions of these disorders.
NA-831, initially showing promising results in phase 2a clinical trials for Alzheimer’s disease, has been identified as a potential treatment for Rett Syndrome and Fragile X due to its neuroprotective and neurogenic properties. These properties are crucial, considering the similarities in brain changes between these conditions and Alzheimer's disease.
Our recent clinical studies on NA-831 for Rett Syndrome and Fragile X have yielded encouraging results. Key findings include:
Based on these outcomes, NA-831 emerges as a promising candidate in the quest for effective treatments for Rett Syndrome and Fragile X.
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