Abstract: Methods of treating a seizure disorder with (1S,3S)-3-amino-4-(difluoromethylidene)cyclopentane-1-carboxylic acid, or (S)-3-amino-4-(difluoromethylenyl)cyclopent-1-ene-1-carboxylic acid (KT-II-115), or a pharmaceutically acceptable salt of any of the preceding, are provided. The methods provide therapeutic compositions that may be used to improve one or more symptoms of a seizure disorder. Methods of treating Prader-Willi syndrome with (1S,3S)-3-amino-4-(difluoromethylidene)cyclopentane-1-carboxylic acid, or (S)-3-amino-4-(difluoromethylenyl)cyclopent-1-ene-1-carboxylic acid (KT-II-115), or a pharmaceutically acceptable salt of any of the preceding, are provided. The methods provide therapeutic compositions that may be used to improve one or more symptoms of Prader-Willi syndrome.

Abstract: Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.

Abstract: Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.

Abstract: Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.

Abstract: Methods of treating tinnitus, acute sensorineural hearing loss, Meniere's disease, Tourette's syndrome, ADHD or addiction with (1S,3S)-3-amino-4-(difluoromethylidene) cyclopentane-1-carboxylic acid or a pharmaceutically acceptable salt thereof are provided. Methods of treating tinnitus, acute sensorineural hearing loss, Meniere's disease, Tourette's syndrome, ADHD or addiction with (S)-3-amino-4-(difluoromethylenyl)cyclopent-1-ene-1-carboxylic acid are provided. Also provided are therapeutic compositions that may be used to improve one or more symptoms of tinnitus, acute sensorineural hearing loss, Meniere's disease, Tourette's syndrome, ADHD or addiction.

Abstract: Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-101 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-128 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-101, pri-miR101 or pre-miR101 to the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-128, pri-miR128 or pre-miR128 to the patient. In embodiments, increased levels of microRNA-101 and/or microRNA-128 cause improvement in one or more symptoms of the seizure disorder.

Abstract: Treatment of non 24 sleep wake disorder using gaboxadol or a pharmaceutically acceptable salt thereof is provided. Pharmaceutical compositions that may be used to improve one or more symptoms of non 24 sleep wake disorder are provided.

Abstract: Methods and compositions for use treating Fragile X syndrome, Angelman syndrome, Fragile X-associated tremor/ataxia syndrome, Autism Spectrum Disorder, Asperger's syndrome, Pervasive developmental disorder not otherwise characterized, Childhood Disintegrative Disorder, Williams syndrome, or Jacobsen syndrome with a compound of Formula 1 as described herein, (S)-3-amino-4-(difluoromethylenyl) cyclopent-1-ene-1-carboxylic acid, (1S,3S)-3-amino-4-(difluoromethylidene) cyclopentane-1-carboxylic acid, or vagabatrin, or pharmaceutically acceptable salts of any of the foregoing, are provided.

Abstract: Use of allosteric modulators and/or gaboxadol for the treatment of epileptic disorders in a subject in need thereof.

Abstract: Methods and pharmaceutical compositions containing gaboxadol or a pharmaceutically acceptable salt thereof for treating 1p36 deletion syndrome are provided.

Abstract: Methods and compositions for treating a metabolic disease include administering gaboxadol or a pharmaceutically acceptable salt thereof to a patient diagnosed with a metabolic disease. In embodiments, administering gaboxadol or a pharmaceutically acceptable salt thereof to a patient diagnosed with a metabolic disease is effective to lower one or more of HbA1c level, fasting plasma glucose level, 2-hour oral glucose tolerance test (OGTT) result level, and random plasma glucose level. Gaboxadol or a pharmaceutically acceptable salt thereof may optionally be administered in combination with a hypoglycemic agent.

Abstract: Methods of treating aggression associated with Alzheimer's Disease including administering gaboxadol or a pharmaceutically acceptable salt thereof to a patient.

Abstract: Expression of micoRNAs that negatively regulate the activity of the SNHG14 gene for can be used in treatment of Angelman Syndrome. Such microRNAs include, for example, MIR-92a and/or MIR-145, as well as analogues and variants thereof, for use in treatment of Angelman Syndrome. Expression vectors such as, for example, AAV vectors may be used to transduce cells for introduction of MIR-92a and/or MIR-145 into target tissues for treatment of Angelman Syndrome.

Abstract: 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.

Abstract: Treatment of Tourette syndrome using gaboxadol or a pharmaceutically acceptable salt thereof is provided. Treatment of tics using gaboxadol or a pharmaceutically acceptable salt thereof is provided. Treatment of stuttering using gaboxadol or a pharmaceutically acceptable salt thereof is provided. Therapeutic compositions that may be used to improve one or more symptoms of Tourette syndrome are provided. Therapeutic compositions that may be used to improve one or more symptoms of tics are provided. Also provided are therapeutic compositions that may be used to improve one or more symptoms of stuttering.

Abstract: Methods of treating developmental disorders and/or seizure disorders with etifoxine, a deuterated analog of etifoxine, or a pharmaceutically acceptable salt, solvate, or prodrug thereof are provided. The methods provide therapeutic compositions that may be used to improve one or more symptoms of the developmental disorder and/or seizure disorder.

Abstract: Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-101 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-128 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-101, pri-miR101 or pre-miR101 to the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-128, pri-miR128 or pre-miR128 to the patient. In embodiments, increased levels of microRNA-101 and/or microRNA-128 cause improvement in one or more symptoms of the seizure disorder.

Abstract: Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-101 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include delivering to the patient an effective amount of a composition that increases the level of microRNA-128 molecules in brain cells of the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-101, pri-miR101 or pre-miR101 to the patient. Methods of treating a seizure disorder in a patient in need thereof are provided which include administering a vector encoding microRNA-128, pri-miR128 or pre-miR128 to the patient. In embodiments, increased levels of microRNA-101 and/or microRNA-128 cause improvement in one or more symptoms of the seizure disorder.

Abstract: Methods and compositions for use treating Fragile X syndrome, Angelman syndrome, Fragile X-associated tremor/ataxia syndrome, Autism Spectrum Disorder, Asperger's syndrome, Pervasive developmental disorder not otherwise characterized, Childhood Disintegrative Disorder, Williams syndrome, or Jacobsen syndrome with a compound of Formula 1 as described herein, (S)-3-amino-4-(difluoromethylenyl) cyclopent-1-ene-1-carboxylic acid, (1S,3S)-3-amino-4-(difluoromethylidene) cyclopentane-1-carboxylic acid, or vagabatrin, or pharmaceutically acceptable salts of any of the foregoing, are provided.

Abstract: Methods and compositions for treating a metabolic disease include administering gaboxadol or a pharmaceutically acceptable salt thereof to a patient diagnosed with a metabolic disease. In embodiments, administering gaboxadol or a pharmaceutically acceptable salt thereof to a patient diagnosed with a metabolic disease is effective to lower one or more of HbA1c level, fasting plasma glucose level, 2-hour oral glucose tolerance test (OGTT) result level, and random plasma glucose level. Gaboxadol or a pharmaceutically acceptable salt thereof may optionally be administered in combination with a hypoglycemic agent.