Eculizumab, a humanized monoclonal antibody against C5 that inhibits terminal complement activation, showed activity in a preliminary 12-week open-label trial in a small cohort of patients with paroxysmal nocturnal hemoglohinuria (PNH). The present study examined whether chronic eculizumab therapy could reduce intravascular hemolysis, stabilize hemoglobin levels, reduce transfusion requirements, and improve quality of life in a double-blind, randomized, placebo-controlled, multi-center global Phase III trial. It has been found that eculizumab stabilized hemoglobin levels, decreased the need for transfusions, and improved quality of life in PNH patients via reduced intravascular hemolysis. Chronic eculizumab treatment appears to be a safe and effective therapy for PNH.

Provided herein are pharmaceutical compositions that comprise myrcene, optionally in admixture with cannabinoids and other terpenes, typically substantially free of THC and THCA, for targeting TRPV1 receptors. Also provided are methods of using the pharmaceutical compositions to desensitize TRPV1 receptors in order to treat pain, cardiovascular diseases such as cardiac hypertrophy, overactive bladder, and chronic cough.

Provided herein are pharmaceutical compositions that comprise myrcene, optionally in admixture with cannabinoids and other terpenes, typically substantially free of THC and THCA, for targeting TRPV1 receptors. Also provided are methods of using the pharmaceutical compositions to desensitize TRPV1 receptors in order to treat pain, cardiovascular diseases such as cardiac hypertrophy, overactive bladder, and chronic cough.

Provided herein is a pharmaceutical composition comprising a compound having the structural formula ##STR00001##
wherein the compound is present in an amount effective to treat or reduce the symptoms of muscular dystrophy. The therapeutically effective amount may be between 10 mg to 200 mg, or may be between 0.01 mg/kg to 10.0 mg/kg. Also provided are methods of treating or reducing the symptoms of muscular dystrophy, comprising the administration, to a patient in need thereof, of a therapeutically effective of the above compound.

Pharmaceutical compositions of the invention comprise functionalized lactone derivatives having a disease-modifying action in the treatment of diseases associated with dysregulation of 5-hydroxytryptamine receptor 7 activity.

The invention provides methods and compositions for treatment of pain, such as joint pain, using capsaicin in a procedure that attenuates transient burning sensation experienced by patients due to capsaicin administration. The methods desirably provide relief from joint pain, such as osteoarthritic knee joint pain, for an extended duration, such as at least about 3 months, 6 months, 9 months, or 1 year. To attenuate the adverse side effect of a transient burning sensation caused by capsaicin-induced neuronal excitation, the methods utilize a cooling article, such as a material wrap cooled via a circulating fluid, to reduce the temperature of tissue to be exposed to capsaicin to within a certain range for certain durations of time, optionally in combination with administering a local anesthetic agent, resulting in the substantial reduction or even elimination of transient burning sensation caused by capsaicin.

The present invention relates to methods for treating uterine hypercontractility disorders. in particular, the present invention relates to methods for treating abnormal uterine bleeding and dysmenorrhea comprising administering to women suffering from such disorders a pharmaceutical composition comprising an S-alkylisothiouronium derivative.

The present invention relates to methods for treating uterine hypercontractility disorders. in particular, the present invention relates to methods for treating abnormal uterine bleeding and dysmenorrhea comprising administering to women suffering from such disorders a pharmaceutical composition comprising an S-alkylisothiouronium derivative.

Provided are topical analgesic spray compositions and topical analgesic spray concentrates containing menthol and camphor in high concentrations. The present disclosure also provides organoleptic compositions for use in the topical analgesic spray compositions and concentrates to provide enhanced sensory experience and long-lasting pain-relief.

Provided are topical analgesic compositions and methods of preparing topical analgesic compositions comprising a stable nano-encapsulating matrix film. The topical analgesic compositions and methods of preparing topical analgesic compositions may be suited for roll-on applicators. Topical analgesic compositions may include 12 to 16 wt. % menthol; 4 to 8 wt. % camphor; 2 to 15 wt. % film-forming polymer; and 50 to 70 wt. % solvent. To be used in a roll-on applicator, the topical analgesic composition has a viscosity from 800 to 1400 centipoise.