Abstract: Provided herein are improved expression constructs for the expression of ATP7B, vectors, and pharmaceutical compositions comprising such constructs. Also provided are methods of treating disease, including, but not limited to, Wilson's Disease.

Abstract: The application relates to nucleic acid regulatory elements (NAREs) that are able to enhance expression of genes in the liver. The application further relates to methods employing NAREs and uses of the NAREs. Expression cassettes and vectors containing NAREs are also disclosed. These are particularly useful for applications using gene therapy.

Abstract: This disclosure provides BDNF expression constructs and vectors comprising such constructs for treating metabolic disorders, including, but not limited to, obesity. The disclosed BDNF gene therapy is more effective than existing BDNF-based gene therapy constructs.

Abstract: The application relates to nucleic acid regulatory elements that are able to enhance expression of genes constitutively in a variety of tissues, or in particular tissues including liver, muscle, and the CNS. The application further relates to methods employing these regulatory elements and uses of these elements. Expression cassettes and vectors containing these nucleic acid regulatory elements are also disclosed. These nucleic acid regulatory elements are particularly useful for applications using gene therapy.

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

Abstract: Pharmaceutical formulations containing gaboxadol or a pharmaceutically acceptable salt thereof and methods of treating essential tremors, Tourette syndrome or Fragile X syndrome are provided. Pharmaceutical formulations herein include transdermal formulations and modified release dosage forms. In embodiments, a modified release dosage form includes an orally disintegrating dosage form. In embodiments, a modified release dosage form includes an extended release dosage form. In embodiments, a modified release dosage form includes a delayed release dosage form. In embodiments, a modified release dosage form includes a pulsatile release dosage form.

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: 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.