Compounds, tautomers and pharmaceutically acceptable salts of the compounds are disclosed, wherein the compounds have the structure of Formula Ia, ##STR00001##
as defined in the specification. Corresponding pharmaceutical compositions, methods of treatment, methods of synthesis, and intermediates are also disclosed.

In one aspect, described herein is an intronic recognition element for splicing modifier (iREMS) that can be recognized by a compound provided herein. In another aspect, described herein are methods for modulating the amount of a product of a gene, wherein a precursor RNA transcript transcribed from the gene contains an intronic REMS, and the methods utilizing a compound described herein. More particularly, described herein are methods for modulating the amount of an RNA transcript or protein product encoded by a gene, wherein a precursor RNA transcript transcribed from the gene comprises an intronic REMS, and the methods utilizing a compound described herein. In another aspect, provided herein are artificial gene constructs comprising an intronic REMS, and uses of those artificial gene constructs to modulate protein production. In another aspect, provided herein are methods for altering endogenous genes to comprise an intronic REMS, and the use of a compound described herein to modulate protein produced from such altered endogenous genes.

Indazole compounds for treating various diseases and pathologies are disclosed. More particularly, the present invention concerns the use of an indazole compound or analogs thereof, in the treatment of disorders characterized by the activation of Wnt pathway signaling (e.g., cancer, abnormal cellular proliferation, angiogenesis, Alzheimer's disease, lung disease and osteoarthritis), the modulation of cellular events mediated by Wnt pathway signaling, as well as genetic diseases and neurological conditions/disorders/diseases due to mutations or dysregulation of the Wnt pathway and/or of one or more of Wnt signaling components. Also provided are methods for treating Wnt-related disease states.

The present invention relates to the use of derivatives between hyaluronic acid, heterocyclic compounds and naturally occurring amino acids in single, oligomeric or polymeric form for the treatment of skeletal diseases, in particular in the regeneration of bone and cartilage tissues.

Described herein are compounds of Formula I:

##STR00001##

and their pharmaceutically acceptable salts, wherein R.sup.1, R.sup.2, R.sup.3, X.sup.1, Y.sup.1, Y.sup.2, Y.sup.3, Y.sup.4 and Y.sup.5 are defined herein; their use as MC4R antagonists; pharmaceutical compositions containing such compounds and salts; the use of such compounds and salts to treat, for example, cachexia, anorexia, or anorexia nervosa; and intermediates and processes for preparing such compounds and salts.

Described herein are compounds of Formula I:

##STR00001##

and their pharmaceutically acceptable salts, wherein R.sup.1, R.sup.2, R.sup.3, X.sup.1, Y.sup.1, Y.sup.2, Y.sup.3, Y.sup.4 and Y.sup.5 are defined herein; their use as MC4R antagonists; pharmaceutical compositions containing such compounds and salts; the use of such compounds and salts to treat, for example, cachexia, anorexia, or anorexia nervosa; and intermediates and processes for preparing such compounds and salts.

The present invention relates to specific doses of and dosing regimens for using a 1,2,4-oxadiazole benzoic acid compound in treating or preventing diseases associated with nonsense mutations. In particular, the invention relates to specific doses and dosing regimens for the use of 3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazol-3-yl]-benzoic acid in mammals having diseases associated with nonsense mutations.

A method of administering a pharmaceutical composition which includes anti-RANKL antibodies, calcium and vitamin D to a patient in need thereof. The method includes subcutaneously administering the anti-RANKL antibodies at a dose of 60-180 mg in 1-3 ml of solution every four weeks, orally administering the calcium at a dose of 400 mg daily, and orally administering the vitamin D at a dose of 800-1200 IU daily. The patient in need thereof has skeletal-related complications due to solid tumors, and the administering of the pharmaceutical composition comprising anti-RANKL antibodies, calcium and vitamin D to the patient in need thereof at least one of treats and prevents hypocalcemia induced by anti-RANKL antibody therapy. The calcium and the vitamin D are provided as a combined single daily dose in a form of effervescent granules, swallowable tablets, swallowable capsules, chewable tablets, or ready-to-use granules.

The present invention relates to an acetylated amphiphilic carbohydrate compound of average molecular weight 1-50 kDa which is based on a glycol chitosan, wherein the levels of acetylation can be modified. The compound can be formulated with hydrophobic compounds such as drugs. The degree of acetylation of the carbohydrate compound is optimised to maximise solubilisation of the drugs. The compound is formulated with drugs and is useful in therapy.

Methods of treating anti-inflammatory conditions through the use of boron-containing small molecules are disclosed.