# US 8,273,715 B2 · Methods of treating fragile X syndrome > Agents: this data is available as JSON from the patentgrep API and MCP server — see https://patentgrep.com/llms.txt - Number: US 8,273,715 B2 - Document ID: US8273715B2 - Status: Granted - Kind: B2 - Application number: 13/362,656 - Filed: 2012-01-31 - Published: 2012-05-17 - Granted: 2012-09-25 - Earliest claimed: 2006-11-22 - Assignees: Seaside Therapeutics, Inc. - Inventors: Kathryn Roberts; Randall L. Carpenter; Mark F. Bear - Web page: https://patentgrep.com/patent/US8273715B2 ## CPC - A61K 31/197: the amino and the carboxyl groups being attached to the same acyclic carbon chain, e.g. gamma-aminobutyric acid [GABA], beta-alanine, epsilon-aminocaproic acid or pantothenic acid (carnitine A61K31/205) - A61K 31/195: having an amino group - A61K 31/215: of carboxylic acids - A61K 31/428: condensed with carbocyclic rings - A61P 15/00: Drugs for genital or sexual disorders (for disorders of sex hormones A61P5/24); Contraceptives - A61P 25/00: Drugs for disorders of the nervous system - A61P 25/08: Antiepileptics; Anticonvulsants - A61P 25/18: Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia - A61P 25/22: Anxiolytics - A61P 43/00: Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 - A61K: PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES (devices or methods specially adapted for bringing pharmaceutical products into particular physical or administering forms A61J3/00; chemical aspects of, or use of materials for deodorisation of air, for disinfection or sterilisation, or for bandages, dressings, absorbent pads or surgical articles A61L; soap compositions C11D) - A61P: SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS ## Abstract Subjects having fragile X syndrome are treated with a composition that includes gamma-aminobutyric acid agonists. The gamma-aminobutyric acid agonist (GABA) can be a GABA(B) agonist, such as baclofen, including racemic and R-baclofen. ## Claims ### Claim 1 A method of improving a social domain in a human having fragile X syndrome, comprising the step of orally administering to the human a composition that includes a therapeutically effective amount of at least one GABA(B) agonist, wherein the GABA(B) agonist is administered to the human in a dose of about 0.001 mg/kg/day to about 5 mg/kg/day. ### Claim 2 (depends on 1) The method of claim 1, wherein the GABA(B) agonist administered to the human includes baclofen. ### Claim 3 (depends on 2) The method of claim 2, wherein the baclofen administered to the human is at least about 70 mole percent R-baclofen, at least about 80 mole percent R-baclofen, at least about 85 mole percent R-baclofen, at least about 90 mole percent R-baclofen, at least about 95 mole percent R-baclofen or at least about 99 mole percent R-baclofen. ### Claim 4 (depends on 3) The method of claim 3, wherein the baclofen administered to the human is at least about 99 mole percent R-baclofen. ### Claim 5 (depends on 1) The method of claim 1, further including administering a Group I mGluR antagonist to the human. ### Claim 6 (depends on 5) The method of claim 5, wherein the mGluR antagonist includes a mGluR5 antagonist. ### Claim 7 (depends on 5) The method of claim 5, wherein the mGluR antagonist includes a mGluR1 member antagonist. ### Claim 8 (depends on 2) The method of claim 2, wherein the baclofen is administered to the human in a dose of at least one member selected from the group consisting of about 2.5 mg, about 5 mg, about 10 mg, about 15 mg, about 20 mg, about 25 mg, about 30 mg, about 40 mg, about 45 mg, about 50 mg and about 100 mg. ### Claim 9 (depends on 8) The method of claim 8, wherein the baclofen is administered in a single daily dose. ### Claim 10 (depends on 8) The method of claim 8, wherein the baclofen is administered to the human in multiple daily doses. ### Claim 11 (depends on 10) The method of claim 10, wherein the baclofen is administered to the human in at least one member selected from the group consisting of two daily doses, three daily doses, four daily doses and five daily doses. ### Claim 12 (depends on 1) The method of claim 1, wherein administering the GABA(B) agonist to the human further causes a decrease in at least one member selected from the group consisting of an obsessive compulsive disorder, a sensory hyperarousal disorder, an anxiety disorder, a seizure disorder, irritability, aggression, startle tremor, repetitive/self-stimulatory behavior, eye diversion, hand biting, hitting of the head, and hitting of others. ### Claim 13 (depends on 1) The method of claim 1, wherein the improvement in the social domain following administration of the gamma-aminobutyric acid GABA(B) agonist to the human includes an increase in at least one member selected from the group consisting of an appropriate response to spoken language, spontaneous attempts to interact, eye contact, motivation, calmness, joint attention and social interactions in therapeutic and educational settings. ### Claim 14 (depends on 1) The method of claim 1, wherein administration of the GABA(B) agonist to the human further improves a communication domain in the human. ### Claim 15 (depends on 1) The method of claim 1, wherein the improvement in the social domain following administration of the GABA(B) agonist to the human further includes an improvement in mood. ### Claim 16 (depends on 1) The method of claim 1, wherein the GABA(B) agonist is administered to the human in a dose of about 0.1 mg/kg/day to about 1 mg/kg/day. ### Claim 17 (depends on 1) The method of claim 1, wherein the GABA(B) agonist is administered to the human in a dose of about 1 mg/kg/day to about 5 mg/kg/day. ### Claim 18 A method of improving a communication domain in a human having fragile X syndrome, comprising the step of orally administering to the human a composition that includes a therapeutically effective amount of at least one GABA(B) agonist, wherein the GABA(B) agonist is administered to the human in a dose of about 0.001 mg/kg/day to about 5 mg/kg/day. ### Claim 19 (depends on 18) The method of claim 18, wherein the GABA(B) agonist administered to the human includes baclofen. ### Claim 20 (depends on 19) The method of claim 19, wherein the baclofen administered to the human is at least about 70 mole percent R-baclofen, at least about 80 mole percent R-baclofen, at least about 85 mole percent R-baclofen, at least about 90 mole percent R-baclofen, at least about 95 mole percent R-baclofen or at least about 99 mole percent R-baclofen. ### Claim 21 (depends on 18) The method of claim 18, wherein the GABA(B) agonist is administered to the human in a dose of about 0.1 mg/kg/day to about 1 mg/kg/day. ### Claim 22 (depends on 18) The method of claim 18, wherein the GABA(B) agonist is administered to the human in a dose of about 1 mg/kg/day to about 5 mg/kg/day. ### Claim 23 A method of improving at least one member selected from the group consisting of a social domain and a communication domain in a human having fragile X syndrome, comprising the step of orally administering to the human a composition that includes a therapeutically effective amount at least about 90 mole percent R-baclofen in the composition, wherein the R-baclofen is administered to the human in a dose of about 0.001 mg/kg/day to about 5 mg/kg/day. ### Claim 24 (depends on 23) The method of claim 23, wherein the baclofen administered to the human is at least about 99 mole percent R-baclofen in the composition. ### Claim 25 (depends on 24) The method of claim 24, wherein the R-baclofen is administered to the human in a dose of at least one member selected from the group consisting of about 2.5 mg, about 5 mg, about 10 mg, about 15 mg, about 20 mg, about 25 mg, about 30 mg, about 40 mg, about 45 mg, about 50 mg and about 100 mg. ### Claim 26 (depends on 25) The method of claim 25, wherein the baclofen is administered to the human in at least one member selected from the group consisting of two daily doses, three daily doses, four daily doses and five daily doses. ### Claim 27 (depends on 23) The method of claim 23, wherein the R-baclofen is administered to the human in a dose of about 0.1 mg/kg/day to about 1 mg/kg/day. ### Claim 28 (depends on 23) The method of claim 23, wherein the R-baclofen is administered to the human in a dose of about 1 mg/kg/day to about 5 mg/kg/day.