Novel Treatment to Ensure Efficacious Dosage for Multiple, Rare Genetic Diseases
Optimal dosing regimen for treating seizures and self-aggressive behavior
Clinicians use a common, FDA approved drug treat symptoms such as seizures and self-aggressive behavior. In some genetically identified patients, too low of a dose is not effective and too high of a dose is not optimal. Suboptimal dosing may also cause sleep problems, dyslipidemia, diabetes, and growth problems. We are working on refining the dosing regimen for patient sub-populations.
Smith Kingsmore Syndrome, Tuberous sclerosis, PMSE, and other similar genetic diseases.
- Repurposing an FDA approved drug.
- A biomarker for optimal dosing.
- Strategy likely applies to other mTORopathies.
John Hogenesch, PhD & Carlos E. Prada, MD, Co-Director, Rasopathy Program