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July 2017 Scleroderma News

CATALYST – for patients with CTD-PAH. CATALYST is a Phase III clinical study evaluating the safety and efficacy of bardoxolone methyl (an oral medication) for the treatment of pulmonary arterial hypertension associated with connective tissue disease (CTD-PAH). Approximately 200 patients will be enrolled at centres in various countries around the world. Reata Pharmaceuticals, Inc., 06/29/2017. (Also see Clinical Trials)

Frequency of motor alterations detected through manometry in patients with esophageal symptoms and scleroderma. Scleroderma is associated with lower esophageal sphincter alterations and symptomatic ineffective esophageal motility. PubMed, Rev Gastroenterol Mex, 2017 Apr - Jun;82(2):193-195. (Also see Dysmotility Syndrome)

Photopheresis (ECP): Advances and Use in Systemic Sclerosis (SSC). Clinical studies have generally demonstrated improvement, especially the skin, in SSc patients receiving ECP and it may be an effective and safe procedure for the treatment of SSc. PubMed, Curr Rheumatol Rep, 2017 Jun;19(6):31. (Also see Scleroderma Treatments– General)

5 Sources of Pain in Scleroderma. Pain, along with fatigue, is one of the main symptoms of scleroderma and one of the toughest to deal with on a day–to–day basis. Scleroderma News, 05/11/2017. (Also see Pain Management)

Survival and quality of life in incident systemic sclerosis–related pulmonary arterial hypertension (SSc-PAH). Despite the significant benefits conferred by advanced PAH therapies suggested in this study, the median survival in SSc PAH remains short at only 4 years. BioMed Central, Arthritis Research & Therapy, 06/02/2017. (Also see Pulmonary Hypertension Prognosis)

Efficacy of mycophenolate mofetil (MMF) and oral cyclophosphamide (CYC) on skin thickness: Post–hoc analyses from the Scleroderma Lung Study I and II. (SLS-I and II) In SLS-II, MMF and CYC resulted in improvements in the modified Rodnan skin score in diffuse cutaneous systemic sclerosis over 24 months. PubMed, Arthritis Care Res (Hoboken), 05/23/2017. (Also see Clinical Trials and Immunosuppressants)

Transethnic meta–analysis identifies GSDMA and PRDM1 as susceptibility genes to systemic sclerosis. GSDMA and PRDM1 are associated with SSc. These findings provide enhanced insight into the genetic and biological basis of Ssc. PubMed, Ann Rheum Dis, 2017 Jun;76(6):1150-1158. (Also see Genetics)

The role of endothelial cells (EC) in the vasculopathy of systemic sclerosis (SSc): A systematic review. This systematic review emphasizes the growing evidence that SSc is primarily a vascular disease where EC dysfunction is present and prominent in different aspects of cell survival. PubMed, Autoimmun Rev, 05/29/2017. (Also see Vascular Involvement)

Circulating Anti–Nuclear Antibodies (ANA) in Systemic Sclerosis (SSc): Utility in Diagnosis and Disease Subsetting. Since these autoantibodies are specifically detected in SSc patients they are widely used in routine clinical practice for diagnosis, clinical subgrouping, and prediction of future organ involvements and prognosis. PubMed, J Nippon Med Sch, 2017;84(2):56-63. (Also see Antibodies)

New tools can potentially play key role in early diagnosis of systemic sclerosis. The results of two studies presented today at the Annual European Congress of Rheumatology (EULAR) 2017 press conference highlight the use of two new tools. News Medical, 06/14/2017. (Also see Nailfold Capillaroscopy and Antibodies)

Fibrocell’s FX-013 Therapy Wins FDA Rare Pediatric Disease Designation for Localized Scleroderma. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to FX-013, a Fibrocell gene therapy candidate to treat children with moderate to severe localized scleroderma. Scleroderma News, 06/13/2017. (Also see Clinical Trials and Juvenile Scleroderma Research)

Go to Scleroderma Medical News: June 2017

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