Friedreich ataxia (FRDA) is caused by a GAA repeat expansion in FXN gene that encodes a mitochondrial protein, frataxin, involved in iron sulfur complex (ISC) assembly. Frataxin deficiency results in abnormal ISC containing proteins, namely respiratory chain complex I-III and aconitases and accumulation of iron in brain and heart of patients. Here, the inventors show that FRDA fibroblasts are unable to limit iron uptake inducing a massive cytosolic iron accumulation and to a lesser extent in mitochondria. The inventors also observed increased transferrin receptor (TfR1) steady state levels and membrane TfR1 accumulation that they ascribed to impaired post-translational modification by palmitoylation as well as delayed transferrin recycling. Finally, the inventors showed that artesunate, dichloroacetate and Coenzyme-A improved TfR1 palmitoylation and thus represent candidate molecules for the treatment of patients with Friedreich ataxia. Thus the present invention relates to methods of treating Friedreich ataxia (FRDA) as well as to methods of predicting whether a patient suffering from FRDA will achieve a therapeutic response.

Provided in the present invention are a compound of Formula (I), a pharmaceutical composition comprising the same, a method for preparing the same, and use thereof as a NS5B polymerase inhibitor, a DNA polymerase inhibitor or a reverse transcriptase inhibitor for the prevention or treatment of viral diseases or cancers.

A new biomaterial is disclosed. The biomaterial is prepared by mixing glycosaminoglycan with a phosphoric acid, phosphate ester, or salt or derivative thereof under the action of a catalyst in a liquid reaction medium at a pH of 2.1 to 4.9. The obtained material is capable of enhancing the immunological resistance in human and in animals, treating diseases in human and in animals caused by viruses or bacteria, strengthening the anti-stress ability of animals, improving the appetite of animals, promoting the growth of animals, inhibiting the growth of tumor, lowering blood fat level, as well as preventing and treating viral diseases in plants.

The present invention discloses a composition of phillyrin and phillygeninin in the preparation of drugs for alleviating or/and treating viral diseases. Experiments prove that the composition of phillyrin and phillygeninin has significant treatment effects on viral influenza and pneumonia, as well as on respiratory syncytial viruses, enteroviruses, herpes zoster simplex viruses, Coxsackie viruses, and the like. This composition is characterized by quick action and less toxic and side effects, and thus is an antiviral drug and health product with safety, high efficiency, stability and simple preparation process, and is suitable for industrial production and easy to promote. The present invention provides a drug, health product and raw material for preventing and treating various viral diseases.

Provided herein are compounds of Formula (I) and pharmaceuticals acceptable salts, solvates, hydrates, polymorphs, co-crystals, tautomers, stereoisomers, and prodrugs thereof. Also provided are pharmaceutical compositions, kits, and methods involving the inventive compounds for the treatment and/or of an infectious disease (e.g., bacterial infection (e.g., P. aeruginosa infection). ##STR00001##

This disclosure relates to certain N4-hydroxycytidine derivatives, compositions, and methods related thereto. In certain embodiments, the disclosure relates to the treatment or prophylaxis of a Zika virus infection.

The present invention relates to a composition comprising a first compound which is a nucleoside analogue capable of inhibiting a bacterial colonisation or infection of a subject; a second compound which is capable of decreasing mitochondrial toxicity of said nucleoside analogue and surprisingly enhance the antibacterial effect of the combination; and a third compound capable of decreasing the concentration in bacteria of nucleosides and/or nucleotides known to compete with nucleoside analogues. The first compound may be AZT, FdUrd, 5-fluorouracil, BrdUrd, IdUrd, didanosine or gemcitabine; the second compound may be uridine or a uridine-comprising compound; and the third compound may be trimethoprim or a compound comprising trimethoprim, such as trimethoprim-sulfa.

The present invention relates to a composition comprising a first compound which is a nucleoside analogue capable of inhibiting a bacterial colonisation or infection of a subject; a second compound which is capable of decreasing mitochondrial toxicity of said nucleoside analogue and surprisingly enhance the antibacterial effect of the combination; and a third compound capable of decreasing the concentration in bacteria of nucleosides and/or nucleotides known to compete with nucleoside analogues. The first compound may be AZT, FdUrd, 5-fluorouracil, BrdUrd, IdUrd, didanosine or gemcitabine; the second compound may be uridine or a uridine-comprising compound; and the third compound may be iclaprim; trimethoprim; or a compound comprising trimethoprim, such as trimethoprim-sulfa.

Certain anti-viral compounds, pharmaceutical compositions, and methods related thereto are disclosed.

Methods of treating infection including sepsis through modulation of a P2X receptor are provided.