Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).

Abstract: The invention relates to mRNA therapy for the treatment of Acute Intermittent Porphyria (AIP). mRNAs for use in the invention, when administered in vivo, encode human porphobilinogen deaminase (PBGD), isoforms thereof, functional fragments thereof, and fusion proteins comprising PBGD. mRNAs of the invention are preferably encapsulated in lipid nanoparticles (LNPs) to affect efficient delivery to cells and/or tissues in subjects, when administered thereto. mRNA therapies of the invention increase and/or restore deficient levels of PBGD expression and/or activity in subjects. mRNA therapies of the invention further decrease levels of toxic metabolites associated with deficient PBGD activity in subjects, namely porphobilinogen and aminolevulinate (PBG and ALA).

Abstract: 5?-methylthioadenosine (MTA) is described as a compound that is susceptible to inhibiting and/or blocking the epithelial-mesenchymal transition (EMT), a process whereby epithelial cells become mesenchymal cells. The periodical intake of MTA [every 24 h for 21 days] significantly improves fibrosis and the markers for hepatic cellular damage in KO-Mdr2 mice with MTA (28 mg/kg). Following the daily oral administration of MTA, both the expression of EMT markers in the total liver and appreciable signs of fibrosis are significantly reduced, indicating the beneficial effect of MTA on the liver affected by the lack of Mdr2. MTA is proposed to be a safe drug, suitable for oral formulation and without secondary effects, to be used in the prevention and/or treatment of diseases associated with EMT, including chronic cholestatic diseases, fibrosis and cholangiocarcinoma.

Abstract: The present invention relates to combinations of 5?-methylthioadenosine and glatiramer acetate, and to their use in the treatment of multiple sclerosis. In a particular embodiment, the present invention relates to a product comprising 5?-methylthioadenosine and glatiramer acetate as a combined preparation for the simultaneous, separate, or sequential use thereof for the prevention and/or treatment of multiple sclerosis.

Abstract: The present invention relates to combinations of 5?-methylthioadenosine and glatiramer acetate, and to their use in the treatment of multiple sclerosis. In a particular embodiment, the present invention relates to a product comprising 5?-methylthioadenosine and glatiramer acetate as a combined preparation for the simultaneous, separate, or sequential use thereof for the prevention and/or treatment of multiple sclerosis.

Abstract: The invention relates to the use of amphiregulin in the production of a medicament which can be used to treat acute hepatic injury and which is administered, for example, in order to: promote a primary endogenous protective reaction in the hepatic tissue against acute hepatic injury, promote DNA synthesis in hepatocytes, prevent the death of hepatocytes in the hepatic tissue of patients with acute hepatic injury, stimulate the regeneration of the remaining hepatic parenchyma following an acute hepatic injury of any aetiology, and stimulate hepatic regeneration following a partial hepatectomy. According to the invention, the amphiregulin is administered as a hepatoprotector medicine for patients with acute hepatic injury of any aetiology and/or as a hepatoprotector medicine and stimulant of hepatocytic regeneration for recipients of a liver transplant from a living donor or a cadaver donor.