Target- |
MechanismAmmonia scavengers |
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Drug Highest PhaseApproved |
First Approval Ctry. / Loc.US |
First Approval Date01 Feb 2013 |
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Drug Highest PhaseApproved |
First Approval Ctry. / Loc.US |
First Approval Date20 Dec 1990 |
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Drug Highest PhaseApproved |
First Approval Ctry. / Loc.US |
First Approval Date21 Feb 1955 |
A Multicenter, Open-label Extension Trial to Evaluate the Efficacy, Safety and Tolerability of HZN-825 in Patients With Diffuse Cutaneous Systemic Sclerosis
Primary Objectives:
1. The primary efficacy objective is to assess the efficacy of 52 weeks of open-label treatment with HZN-825 in participants with diffuse cutaneous systemic sclerosis, as measured by change from both baselines in forced vital capacity percent (FVC %) predicted.
2. The primary safety objective is to examine the safety and tolerability of 52 weeks of open-label treatment with HZN-825, inclusive of, but not limited to, adverse events (AEs), serious AEs (SAEs) and the adverse event of special interest (AESI), from Day 1 to 4 weeks after last dose.
Assessing the Role of the NLRP3 Inflammasome in Driving Inflammation in Affected Joints of Patients With Intercritical Gout
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.
A Double Blind, Placebo Controlled, Dose Escalation Trial of Glycerol Phenylbutyrate Corrector Therapy for Cystic Fibrosis
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.
100 Clinical Results associated with Horizon Pharma Ireland Ltd.
0 Patents (Medical) associated with Horizon Pharma Ireland Ltd.
100 Deals associated with Horizon Pharma Ireland Ltd.
100 Translational Medicine associated with Horizon Pharma Ireland Ltd.