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: 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 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: 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: Methods of treating a seizure disorder with (1S,3S)-3-amino (difluoromethylidene)cyclopentane-1-carboxylic acid, or (S)-3-amino (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: 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: Methods of increasing tonic inhibition in a subject in need thereof, for example a subject with Fragile X syndrome or Angelman syndrome are disclosed. Methods of treating secondary insomnia in a subject with a neurodegenerative disease or disorder are also disclosed.

Abstract: Disclosed are methods for treating neurological disorders such as Parkinson's disease (PD) using glutamic acid decarboxylase (GAD) and identifying PD patients that will be most receptive to the method of treating PD. In one aspect, the disclosure provides a method of treating PD in a subject in need thereof, the method comprising: (a) identifying a subject having less than about 10 hours, and preferably less than about 8 hours, of on-time per day; and (b) administering to the subject a composition comprising a therapeutically effective amount of one or more vectors to the subthalamic nucleus of the patient, wherein each vector comprises a nucleic acid sequence encoding glutamic acid decarboxylase (GAD) and wherein the subject's on-time is increased.

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 using gaboxadol or a pharmaceutically acceptable salt thereof and compositions of gaboxadol or a pharmaceutically acceptable salt thereof for use in treating a substance use disorder, preventing a substance use craving, diminishing a substance use craving, and/or facilitating substance use cessation are provided. Substance use disorders include alcohol use disorder, caffeine use disorder, cannabis use disorder, hallucinogen use disorder, inhalant use disorder, opioid use disorder, sedative use disorder, stimulant use disorder, tobacco use disorder and nicotine use disorder.

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: Use of gaboxadol, allopregnanolone, or ganaxolone, or a pharmaceutical salt of any of the foregoing, for the treatment of cervical dystonia, multiple system atrophy, myoclonus, progressive supranuclear palsy, restless legs syndrome, Wilson's disease, ataxia, chorea, or dystonia in a subject in need thereof.

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.