Horizon Therapeutics Ireland DAC

Gout is an autoinflammatory disease characterized by flares of painful joint inflammation. This inflammation occurs in response to uric acid that crystallizes. After a gout attack, patients usually enter a period that is accompanied by low grade inflammation but is otherwise relatively asymptomatic. Gout is typically associated with certain markers, and this study is going describe specific markers in patients that are in between gout attacks. Research has been focused on studying this phase between gout attacks in hopes to manage and prevent the onset of future gout attacks. Biopsies will be taken from the affected joint and blood will be drawn from patients who are currently in between gout attacks. This work will provide important information regarding how crystals in the joint lining are associated with chronic inflammation in the periods between gout attacks. Moreover, this study will identify novel biomarkers that may be useful in determining the severity of disease activity through a blood test.

We propose to test the effectiveness of the combination of CF pancreatic enzyme replacement therapy (PERT) on absorption of Ravicti® and subsequent restoration of nasal epithelial cystic fibrosis transmembrane conductance regulator (CFTR)-mediated chloride transport during the nasal potential difference (NPD) test. Funding source FDA Office of Orphan Products Development.

This purpose of this study is to evaluate the safety and to find the optimal dose in participants with human epidermal growth factor receptor 2 (HER2) positive breast cancer who are given the combination of Interferon-gamma with paclitaxel, trastuzumab and pertuzumab. This study will also look at other effects of Interferon-gamma with paclitaxel, trastuzumab and pertuzumab, including its effect on this type of cancer.
Interferon-gamma is a biologically manufactured protein that is similar to a protein the body makes naturally. In the body, interferon gamma is produced by immune cells and helps to prevent serious infections.