The present invention relates to vectors and compositions for the treatment of glycogen storage disease III.

The present disclosure provides technologies relating to lysosomal activation. The disclosure provides several strategies for increasing level and/or activity of lysosomal enzyme, and furthermore demonstrates the surprising applicability of such strategies in the treatment and/or prophylaxis of certain proteinopathies. Among other things, the present invention provides methods and compositions for the treatment and/or prophylaxis of proteinopathies other than lysosomal storage diseases through lysosomal activation. In particular, the present disclosure provides methods and compositions for the treatment and/or prophylaxis of neurodegenerative proteinopathies, and in particular those associated with accumulation of α-synuclein. The present disclosure specifically provides methods and compositions for the treatment and/or prophylaxis of Parkinson's disease.

This disclosure features compositions, methods, and kits for the treatment of metabolic disorders such as diabetes and obesity.

This disclosure features compositions, methods, and kits for the treatment of metabolic disorders such as diabetes and obesity.

The present invention provides novel compounds comprising cannabidiolic acid (CBDA) derivatives, including acids and esters, and uses thereof in the treatment of various diseases, disorders, conditions and symptoms.

The present invention provides uses of a glucokinase activator in combination with metformin. Uses include treating type 2 diabetes, lowering blood glucose, improving insulin sensitivity, enhancing phosphorylation of glucose, and improving the therapeutic effectiveness of metformin. The invention also provides pharmaceutical compositions that comprise a GK activator and metformin. The invention also provides a salt formed between metformin and a GK activator.

An injection device adapted to be worn on the body of a patient. The device includes an injector containing an agent for treating hypoglycaemia. A control assembly including a signal receiver is configured to receive signals from a glucose sensor, the control assembly being electrically coupled to an alarm and configured to activate the alarm upon a signal received in the signal receiver being indicative of hypoglycaemia, the control assembly being configured to cause the injector to administer through a needle a dose of the agent to the patient after a predetermined period of time of the signal receiver receiving the signal indicative of hypoglycaemia, the control assembly having a manually activated switch that when activated prior to the expiration of the predetermined period of time causes the control assembly to abort the injection of the agent.

The application relates to compounds of formula A:

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or a salt, solvate, ester, tautomer, amino acide conjugate, or metabolite thereof. The compounds of formula A are TGR5 modulators useful for the treatment of various diseases, including metabolic disease, inflammatory disease, autoimmune disease, cardiac disease, kidney disease, cancer, and gastrointestinal disease.

The application relates to compounds of formula A:

##STR00001##

or a salt, solvate, ester, tautomer, amino acide conjugate, or metabolite thereof. The compounds of formula A are TGR5 modulators useful for the treatment of various diseases, including metabolic disease, inflammatory disease, autoimmune disease, cardiac disease, kidney disease, cancer, and gastrointestinal disease.

The present disclosure relates generally to compounds which bind to the NR1H4 receptor (FXR) and act as agonists of FXR. The disclosure further relates to the use of the compounds for the preparation of a medicament for the treatment of diseases and/or conditions through binding of said nuclear receptor by said compounds and to a process for the synthesis of said compounds.