Methods of treating a disease characterized by mRNA instability or nonsense-mediated decay of an mRNA of a disease-associated gene in a subject by administering a pharmaceutical composition comprising at least one agent that decreases FTO expression, function or both are provided. Kits and pharmaceutical compositions comprising an agent that decreases FTO expression, function or both and a read-through promoting agent are also provided, as are methods of determining suitability of a subject to be treated with an agent that decreases FTO expression, function or both.

The present invention relates to agents and methods for treating gastrointestinal cancer (e.g., metastatic colorectal cancer) in a subject in need thereof. The method includes suppressing the enzymatic activity of DHODH and/or decreasing the level of creatine via suppression of creatine transporter channel SLC6a8 in the subject. In some embodiments, the suppression step can be carried out by administering to the subject a set of small molecule compounds.

The present invention pertains to: the provision of a method for promoting skin wound healing and a composition to be used for promoting skin wound healing; the provision of a method useful for preventing or ameliorating ulcers and bedsores or a composition therefor; the provision of a method for preventing skin aging and a composition to be used for preventing skin aging; the provision of a composition useful for preventing or ameliorating skin damage caused by an anticancer agent; and, furthermore, the provision of a method for enhancing the regeneration ability of epidermal stem cells and a composition for enhancing the regeneration ability of epidermal stem cells. More specifically, use of a composition, said composition being characterized by comprising, as an active ingredient, a substance selected from the group consisting of a substance which induces or maintains the expression of COL17A1 in cells, a substance which inhibits the degradation of COL17A1 in cells, a substance which promotes the competitive amplification ability of epidermal stem cells, a substance which suppresses genomic stress or oxidative stress in cells and a substance which prevents DNA damage in cells, enables the promotion of skin wound healing, protection from an anticancer agent and prevention of skin aging. Thus, the regeneration ability of epidermal stem cells can be enhanced.

The invention provides anelastase inhibitor for use in the promotion of muscle regeneration in the treatment of a myopathy, as well as a method for promoting muscle regeneration in a subject with a myopathy, the method comprising providing the subject with a therapeutically effective amount of an elastase inhibitor. Further provided is a pharmaceutical composition comprising anelastase inhibitor for use in the promotion of muscle regeneration in the treatment of a myopathy. Elastase inhibitors may have a protective effect on muscle progenitor cells and their regenerative potential, which aids muscle cell regeneration. By protecting regenerative potential of muscle progenitor cells, elastase inhibitors enable or enhance the gown of new or existing muscle fibres.

Inhibitors of MAPK3 (ERK1 MAP kinase), in particular polypeptides having the ability to stimulate the global ERK signalling pathway in the brain and their use as neuroprotective and/or cognitive enhancing agents, are disclosed. Related polynucleotides, vectors, host cells and pharmaceutical compositions able to inhibit MAPK3, causing the stimulation of the global ERK signalling pathway, are also disclosed. Additionally, use of the afore inhibitors or stimulators in the treatment of neurodegenerative or neuropsychiatric disorders and cognitive impairment is also disclosed.

Compositions are provided for the liquid oral administration of topiramate and its salts. The invention further provides methods for treating diseases and disorders using the compositions.

The present invention is inter alia concerned with a combination (i) a first compound selected from the group consisting of a norepinephrine-dopamine reuptake inhibitor (NDRI), a catecholamine and pharmaceutically acceptable salts thereof, (ii) a second compound selected from the group consisting of vitamin D3, calcifediol, calcitriol, vitamin D2, ercalcidiol and ercalcitriol, and (iii) a third compound selected from the group consisting of vitamin K1, vitamin K2 and vitamin K3, for use in the treatment of an inflammatory disease, preferably multiple sclerosis.

An aortic aneurysm has few noticeable symptoms and is at high risk for rupture without a sign of abnormality. Further, if it ruptures, it causes a shock state due to massive blood loss and results in a very low survival rate. Thus, when the aortic aneurysm is found by physical examination or the like, a surgery with a stent graft or the like needs to be performed according to the state of its progression.

A pharmaceutical composition for preventing an aortic aneurysm including a medium chain fatty acid as a main component has an effect of inhibiting the occurrence and progression of the aortic aneurysm. Thus, when the aortic aneurysm is found, the composition through continuous intake can exhibit effects including inhibition of the progression of the aortic aneurysm and improvement in the vital prognosis.

A composition used for combating metabolic diseases and uses of (or a method for) the composition. The pharmaceutical composition comprises therapeutic agent A or a pharmaceutically acceptable salt thereof; therapeutic agent B or a pharmaceutically acceptable salt thereof; and at least one pharmaceutically acceptable excipient, where therapeutic agent A is a non-steroidal anti-inflammatory medicament, and therapeutic agent B is a fatty acid oxidation inhibitor. The pharmaceutical composition effectively treats or prevents obesity, non-alcoholic fatty liver, polycystic ovary syndrome, type 2 diabetes, and metabolic syndrome diseases caused by insulin resistance.

The present invention provides solid forms of an MK2 inhibitor, compositions thereof, and methods of using the same.