The invention is directed to a method of treating attention-deficit/hyperactivity disorder (ADHD) in a subject, comprising administering a therapeutically effective amount of a carbamoyl compound, or pharmaceutically acceptable salt thereof.

New compounds of formula I are described:

##STR00001##

The compounds have potentially valuable pharmaceutical properties in the treatment of some central and peripheral nervous system disorders.

Provided herein are multicyclic compounds, methods of their synthesis, pharmaceutical compositions comprising the compounds, and methods of their use. The compounds provided herein are useful for the treatment, prevention, and/or management of various neurological disorders, including but not limited to, psychosis and schizophrenia.

The present invention describes novel nanoparticle compositions, and systems and methods utilizing them for treating disorders and/or conditions. Methods generally involve administering nanoparticle compositions (e.g., nanoparticle compositions comprising at least one known therapeutic agent and/or independently active biologically active agent; and/or empty nanoparticle compositions) to a subject in need thereof.

A pharmaceutical liquid composition includes a solution of a physiologically acceptable salt of 4-phenylbutyric acid, which in one embodiment is the sodium salt of 4-phenylbutyric acid, in an aqueous medium at a concentration of at least 1.34 mmol/ml. The aqueous medium includes glycerol; a viscosity enhancing agent, where the viscosity enhancing agent includes a cellulose derivate, and water. The pharmaceutical liquid composition further includes a sweetening agent.

Methods of treating subjects having G4C2 dipeptide repeat expansion in the gene C9ORF72, including subjects having amyotrophic lateral sclerosis or frontotemporal degeneration (ALS/FTD), are provided. Compounds directed at reducing the toxicity of G4C2 dipeptide repeat expansion in the gene C9ORF72 are described.

Methods of treating subjects having G4C2 dipeptide repeat expansion in the gene C9ORF72, including subjects having amyotrophic lateral sclerosis or frontotemporal degeneration (ALS/FTD), are provided. Compounds directed at reducing the toxicity of G4C2 dipeptide repeat expansion in the gene C9ORF72 are described.

The invention concerns a trans-splicing RNA (tsRNA) molecule comprising one or multiple unstructured binding domains; a cell or vector comprising said tsRNA; and a method for killing cells or treating a disease using said tsRNA.

The invention concerns a trans-splicing RNA (tsRNA) molecule comprising one or multiple unstructured binding domains; a cell or vector comprising said tsRNA; and a method for killing cells or treating a disease using said tsRNA.

Methods of treating a subject having or at risk of developing a neurodegenerative disease or condition associated with demyelination, insufficient myelination, or underdevelopment of myelin sheath are described. The methods include administration of a therapeutically effective amount of sobetirome, or a pharmaceutically acceptable salt thereof.