Compositions and methods for reducing tactile dysfunction, anxiety, and social impairment
The present invention provides novel peripherally-restricted benzodiazepines with reduced blood brain barrier permeability and methods of use thereof for reducing tactile dysfunction, social impairment, and anxiety in a subject diagnosed with Autism Spectrum Disorder, Rett syndrome, Phelan McDermid syndrome, or Fragile X syndrome.
1. A compound having the structure:
or a pharmaceutically acceptable salt thereof.
2. A pharmaceutical composition comprising the compound or pharmaceutically acceptable salt according to claim 1 , and a pharmaceutically acceptable excipient.
3. A method of reducing tactile dysfunction in a human subject diagnosed with Autism Spectrum Disorder (ASD), Rett Syndrome (RTT), Phelan McDermid syndrome (PMS), or Fragile X Syndrome, by administering to the subject the compound or pharmaceutically acceptable salt of claim 1 in an amount and for a duration sufficient to reduce the tactile dysfunction.
4. A method of reducing anxiety or social impairment in a human subject diagnosed with ASD, RTT, PMS, or Fragile X Syndrome by administering to the subject the compound or pharmaceutically acceptable salt of claim 1 in an amount and for a duration sufficient to reduce the anxiety or social impairment.
5. The compound of claim 1 having the structure:
or a pharmaceutically acceptable salt thereof.
6. The compound of claim 1 having the structure:
or a pharmaceutically acceptable salt thereof.
7. The compound of claim 1 having the structure:
or a pharmaceutically acceptable salt thereof.
8. The pharmaceutical composition of claim 2 , wherein the compound has the structure:
or a pharmaceutically acceptable salt thereof.
9. The pharmaceutical composition of claim 2 , wherein the compound has the structure:
or a pharmaceutically acceptable salt thereof.
10. The pharmaceutical composition of claim 2 , wherein the compound has the structure:
or a pharmaceutically acceptable salt thereof.