title: Seeking approval: present and future therapies for pemphigus vulgaris.
abstract: Pemphigus vulgaris is an autoimmune blistering disease of the skin and mucous membranes. Despite the potentially fatal prognosis, there are currently no FDA-approved treatments specifically for pemphigus. In 2006, the FDA designated orphan drug status to mycophenolate mofetil for the treatment of pemphigus vulgaris, indicating both federal and commercial interest in developing therapies for this devastating disease. This review focuses on pemphigus therapies that are currently in preclinical or clinical trials, as well as potential novel therapies based on recent advances in the understanding of the pathophysiology of this disease.
authors.affiliation: University of Pennsylvania, Department of Dermatology, 217A Clinical Research Building, 415 Curie Boulevard, Philadelphia, PA 19104, USA.
authors.forename: Xuming
authors.initials: X
authors.lastname: Mao
authors.forename: Aimee S
authors.initials: AS
authors.lastname: Payne
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D000818:Animals; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D058846:Antibodies, Monoclonal, Murine-Derived; D001323:Autoantibodies / Q000276:immunology; D019013:CD40 Antigens / Q000037:antagonists & inhibitors; D023201:CD40 Ligand / Q000037:antagonists & inhibitors; D018679:Cholinergic Agonists / Q000627:therapeutic use; D004359:Drug Therapy, Combination; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000627:therapeutic use; D007166:Immunosuppressive Agents / Q000627:therapeutic use; D009173:Mycophenolic Acid / Q000031:analogs & derivatives / Q000627:therapeutic use; D010392:Pemphigus / Q000175:diagnosis* / Q000628:therapy*; D010956:Plasmapheresis; D000069283:Rituximab; D014409:Tumor Necrosis Factor-alpha / Q000037:antagonists & inhibitors; D048051:p38 Mitogen-Activated Protein Kinases / Q000037:antagonists & inhibitors
chemical_list: D000305:Adrenal Cortex Hormones; D000911:Antibodies, Monoclonal; D058846:Antibodies, Monoclonal, Murine-Derived; D001323:Autoantibodies; D019013:CD40 Antigens; D018679:Cholinergic Agonists; D016756:Immunoglobulins, Intravenous; D007166:Immunosuppressive Agents; D014409:Tumor Necrosis Factor-alpha; D023201:CD40 Ligand; D000069283:Rituximab; D048051:p38 Mitogen-Activated Protein Kinases; D009173:Mycophenolic Acid