Refined uses of gaba a receptor modulators in treatment of fragile x syndrome
AssigneeChildren's Hospital Medical Center
InventorsCraig A. ERICKSON, Christina GROSS, Ernest PEDAPATI, Lauren SCHMITT
Life of this patent
Estimating the term…
Abstract
Identification of Fragile X syndrome (FXS) patients who are likely to respond to a treatment involving a GABAA receptor modulator (e.g., a selective GABAA receptor stimulator), for example, at a low dose, using peripheral Fragile X mental retardation protein (FMRP) as a biomarker, either taken alone or in combination with other biomarkers. Also provided here are methods for treating such FXS patients using the GABAA receptor modulator (e.g., a selective GABAA receptor stimulator at a low dose).
Claims28
A method for identifying a Fragile X Syndrome (FXS) patient who is likely to respond to a treatment for FXS (FXS treatment), the method comprising:
- (a) providing a biological sample of a FXS patient;
- (b) detecting Fragile X mental retardation protein (FMRP) in the biological sample;
- and
- (c) identifying the FXS patient for the FXS treatment, wherein the FMRP is undetectable in the biological sample or has a level no greater than a predetermined value.
A method for treating Fragile X syndrome (FXS), comprising subjecting an FXS patient to a treatment for FXS (FXS treatment), wherein the FXS patient has undetectable peripheral Fragile X mental retardation protein (FMRP) or a level of peripheral FMRP no greater than a predetermined value.
A pharmaceutical composition for use in treating Fragile X syndrome (FXS) in a FXS patient, wherein the pharmaceutical composition comprises a GABAA modulator, a muscle relaxer, and/or a vector expressing a functional FMRP polypeptide, and wherein the FXS patient has undetectable peripheral Fragile X mental retardation protein (FMRP) or a level of peripheral FMRP no greater than a predetermined value.
Dependencies are inferred from the claim text.