METHODS OF TREATING DEVELOPMENTAL DISORDERS WITH GABOXADOL
Methods of treating developmental disorders such as Angelman syndrome, Fragile X syndrome, Fragile X-associated tremor/ataxia syndrome (FXTAS), Autistic Spectrum Disorder, Autism, Asperger's syndrome, pervasive developmental disorder, Childhood Disintegrative Disorder, Rett syndrome, Lanau-Kleffner Syndrome, Prader-Willi Syndrome, Tardive Dyskinesia, and/or Williams Syndrome with gaboxadol or a pharmaceutically acceptable salt thereof are provided. The methods provide therapeutic compositions that may be used to improve one or more symptoms of the developmental disorder.
1 . A method of treating a developmental disorder selected from the group consisting of an Autistic Spectrum Disorder, Autism, Angelman syndrome, and Fragile X syndrome comprising administering to a patient in need thereof gaboxadol or a pharmaceutically acceptable salt thereof, wherein the method provides improvement in one or more symptoms of the developmental disorder in the patient for more than 6 hours after administration.
2 . The method of claim 1 , wherein the patient is administered about 1 mg to about 30 mg gaboxadol or a pharmaceutically acceptable salt thereof.
3 . The method of claim 1 , wherein the patient is administered about 1 mg to about 25 mg gaboxadol or a pharmaceutically acceptable salt thereof.
4 . The method of claim 1 , wherein the patient is administered about 1 mg to about 15 mg gaboxadol or a pharmaceutically acceptable salt thereof.
5 . The method of claim 1 , wherein the patient is administered about 1 mg to about 10 mg gaboxadol or a pharmaceutically acceptable salt thereof.
6 . The method of claim 1 , wherein the in vivo plasma profile of the patient 6 hours after administration of the gaboxadol or pharmaceutically acceptable salt thereof is reduced by more than 50%.
7 . The method of claim 1 , wherein the AUC 6-12 of the patient 6 hours after administration of the gaboxadol or pharmaceutically acceptable salt thereof is less than 75% of the administered dose.
8 . The method of claim 1 , wherein the method provides improvement in the patient for more than 8 hours.
9 . The method of claim 1 wherein the method provides improvement in the patient for at least 12 hours.
10 . The method of claim 1 , wherein the method provides improvement in at least one symptom selected from the group consisting of ataxia, gait, speech impairment, vocalization, cognition, motor activity, clinical seizure, hand flapping, and short attention span
11 . The method of claim 1 wherein the method provides improvement in the patient for more than 12 hours after administration.
12 . A method of treating a developmental disorder selected from the group consisting of an Autistic Spectrum Disorder, Autism, Angelman syndrome, and Fragile X syndrome comprising administering to a patient in need thereof gaboxadol or a pharmaceutically acceptable salt thereof wherein the method provides an in vivo plasma profile comprising a C max less than about 400 ng/ml and wherein the method provides improvement in next day functioning of the patient.
13 . A method of treating a developmental disorder selected from the group consisting of an Autistic Spectrum Disorder, Autism, Angelman syndrome, and Fragile X syndrome comprising administering to a patient in need thereof gaboxadol or a pharmaceutically acceptable salt thereof wherein the method provides an in vivo plasma profile comprising a AUC 6-12 of less than about 900 ng·hr/ml and wherein the method provides improvement in next day functioning of the patient.