The present invention includes a low dose and sustained release formulation of a mitochondrial uncoupling agent The compositions of the invention are useful for preventing or treating a disease or disorder, such as non-alcoholic fatty liver disease, non-alcoholic steatohepatitis, insulin resistance and/or diabetes, in a subject in need thereof.

Disclosed herein are methods for improving exosome production or stimulating exosome secretion by cells. The methods comprise inhibiting at least one step of the glycolytic pathway by contacting the cells with at least one glycolytic inhibitor and/or inhibiting mitochondrial function by contacting the cells with at least one inhibitor of mitochondrial function.

Disclosed herein are methods for improving exosome production or stimulating exosome secretion by cells. The methods comprise inhibiting at least one step of the glycolytic pathway by contacting the cells with at least one glycolytic inhibitor and/or inhibiting mitochondrial function by contacting the cells with at least one inhibitor of mitochondrial function.

A composition and method of treatment of neuromuscular, neuromuscular degenerative, neurodegenerative, autoimmune, developmental, traumatic, hearing loss related, and/or metabolic diseases, including spinal muscular atrophy (SMA) syndrome (SMA1, SMA2, SMA3, and SMA4, also called Type I, II, III and IV), traumatic brain injury (TBI), concussion, keratoconjunctivitis sicca (Dry Eye Disease), glaucoma, Sjogren's syndrome, rheumatoid arthritis, post-LASIK surgery, anti-depressants use, Wolfram Syndrome, and Wolcott-Rallison syndrome. The composition is selected from the group consisting of 2,3-DNP, 2,4-DNP, 2,5-DNP, 2,6-DNP, 3,4-DNP, or 3,5-DNP, bipartite 2,3-dinitrophenol, 2,4-dinitrophenol, 2,5-dinitrophenol, 2,6-dinitrophenol, 3,4-dinitrophenol, or 3,5-dinitrophenol (2,3-DNP, 2,4-DNP, 2,5-DNP, 2,6-DNP, 3,4-DNP, or 3,5-DNP) prodrugs; Gemini prodrugs, bioprecursor molecules, and combinations thereof. A dose of the composition for treatment of neurodegenerative diseases may be from about 0.01 mg/kg of body weight to about 50 mg/kg of body weight of the patient in need of treatment. A dose of the composition for treatment of metabolic diseases may be from about 1 mg/70 kg of body weight to about 100 mg/70 kg of body weight of the patient in need of treatment, and a maximum dose per day is about 200 mg/70 kg of body weight of the patient in need of treatment.

A composition and method of treatment of neuromuscular, neuromuscular degenerative, neurodegenerative, autoimmune, developmental, traumatic, hearing loss related, and/or metabolic diseases, including spinal muscular atrophy (SMA) syndrome (SMA1, SMA2, SMA3, and SMA4, also called Type I, II, III and IV), traumatic brain injury (TBI), concussion, keratoconjunctivitis sicca (Dry Eye Disease), glaucoma, Sjogren's syndrome, rheumatoid arthritis, post-LASIK surgery, anti-depressants use, Wolfram Syndrome, and Wolcott-Rallison syndrome. The composition is selected from the group consisting of 2,3-DNP, 2,4-DNP, 2,5-DNP, 2,6-DNP, 3,4-DNP, or 3,5-DNP, bipartite 2,3-dinitrophenol, 2,4-dinitrophenol, 2,5-dinitrophenol, 2,6-dinitrophenol, 3,4-dinitrophenol, or 3,5-dinitrophenol (2,3-DNP, 2,4-DNP, 2,5-DNP, 2,6-DNP, 3,4-DNP, or 3,5-DNP) prodrugs; Gemini prodrugs, bioprecursor molecules, and combinations thereof. A dose of the composition for treatment of neurodegenerative diseases may be from about 0.01 mg/kg of body weight to about 50 mg/kg of body weight of the patient in need of treatment. A dose of the composition for treatment of metabolic diseases may be from about 1 mg/70 kg of body weight to about 100 mg/70 kg of body weight of the patient in need of treatment, and a maximum dose per day is about 200 mg/70 kg of body weight of the patient in need of treatment.

The present disclosure relates to certain (2S)—N-[(1S)-1-cyano-2-phenylethyl]-1,4-oxazepane-2-carboxamide compounds (including pharmaceutically acceptable salts thereof), ##STR00001##
that inhibit dipeptidyl peptidase 1 (DPP1) activity, to their utility in treating and/or preventing clinical conditions including respiratory diseases, such as asthma and chronic obstructive pulmonary disease (COPD), to their use in therapy, to pharmaceutical compositions containing them and to processes for preparing such compounds.

Embodiments disclosed herein provide methods for treating neoplasm in a mammal, comprising intratumorally administering to the neoplasm an effective amount of a pharmaceutical composition comprising: a hapten; and a redox agent, whereby the neoplasm is treated.

Embodiments disclosed herein provide methods for treating neoplasm in a mammal, comprising intratumorally administering to the neoplasm an effective amount of a pharmaceutical composition comprising: a hapten; and a redox agent, whereby the neoplasm is treated.

Provided are methods of treating a neurological or psychiatric disorder in a subject in need thereof, comprising: administering to the subject a therapeutically effective amount of ketamine or a pharmaceutically acceptable salt thereof in combination with an amount of one or more compounds effective to increase the level of nicotinamide adenine dinucleotide (NAD.sup.+) in the subject.

Provided are methods of treating a neurological or psychiatric disorder in a subject in need thereof, comprising: administering to the subject a therapeutically effective amount of ketamine or a pharmaceutically acceptable salt thereof in combination with an amount of one or more compounds effective to increase the level of nicotinamide adenine dinucleotide (NAD.sup.+) in the subject.