Abstract: Disclosed is a method of treatment of a disease associated with abnormal protein aggregation comprising parenterally administering pharmaceutical formulations comprising trehalose. Also disclosed is an injectable aqueous pharmaceutical formulation comprising a therapeutically effective amount of trehalose.
Abstract: The present invention provides intravenous compositions of trehalose for the treatment of signs and symptoms of spinocerebellar ataxia (SCA).
Abstract: The present invention provides intravenous compositions of trehalose for the treatment of signs and symptoms of oculopharyngeal muscular dystrophy (OPMD).
Abstract: The invention provides fusion protein constructs comprising a functional mitochondrial protein and methods of treating mitochondrial disorders by the fusion proteins and compositions thereof.