The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and NORD’s founding at a dinner tonight in Washington, D.C. The 2018 Rare Impact Awards, to be held at the Andrew W. Mellon Auditorium, will be webcast via Facebook for those…
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Clinical features of sarcoidosis-associated pulmonary hypertension are similar across patients in the U.S., Europe and Middle East, but treatment is more likely to be given to patients outside the United States, a study reports. These findings were drawn from data on 176 sarcoidosis-associated pulmonary hypertension (SAPH) patients enrolled in a…
Scottish patients with pulmonary arterial hypertension (PAH) can now be treated with Actelion‘s Uptravi (selexipag) through the country’s National Health System (NHS) after a positive recommendation from the Scottish Medicines Consortium (SMC). However, the use of this therapy will be restricted to adult patients in WHO functional…
Reata Pharmaceuticals is currently recruiting participants for its Phase 3 CATALYST trial assessing the effectiveness of bardoxolone methyl in connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH). After analysis of the Phase 2 LARIAT study (NCT02036970) results in pulmonary hypertension (PH) and interim data of the first 100…
A project investigating the role of a special protein called PFKFB3 in pulmonary hypertension (PH) has been awarded a $2.2 million grant from the National Heart, Lung and Blood Institute. PFKFB3 is a potential new therapeutic target for early stages of PH. PFKFB3 is an enzyme involved in the…
Bellerophon Therapeutics announced that is moving forward with a planned Phase 2b trial that will investigate INOpulse as a therapy for pulmonary hypertension associated with chronic obstructive pulmonary disease (PH-COPD), following an agreement with the U.S. Food and Drug Administration regarding the study’s design. INOpulse therapy is based on…
Age, ischemic heart disease, and kidney dysfunction are important predictors of outcomes in patients with idiopathic pulmonary arterial hypertension (IPAH), a Swedish study shows. The research, “Impact of age and comorbidity on risk stratification in idiopathic pulmonary arterial hypertension,” was published recently in the European Respiratory Journal.
A gene called FoxM1 was seen to be an important regulator of the vascular remodeling process involved in pulmonary hypertension. It triggers the proliferation of smooth muscle cells in the lung’s blood vessels, which in turn promotes the thickening of artery walls and elevated arterial blood pressure. Inhibiting or blocking FoxM1 activity…
At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…
Patients with pulmonary arterial hypertension (PAH) who can walk more than 400 meters (437 yards) on a six-minute walk test have better long-term prognosis, with lower risk of PAH-related death or hospitalization, results from a Phase 3 clinical trial show. The study, “…
Activation of a molecule called PPAR-gamma may be a future strategy to prevent pulmonary arterial hypertension (PAH) and other cardiovascular/pulmonary diseases, a study suggests. Findings were reported in the study, “PPARγ agonist pioglitazone reverses pulmonary hypertension and prevents right heart failure via fatty acid oxidation,” published in…
Actelion Pharmaceuticals submitted a supplemental new drug application to U.S. regulatory authorities seeking expanded approval for Opsumit (macitentan) to treat inoperable chronic thromboembolic pulmonary hypertension (CTEPH). The U.S. Food and Drug Administration application is based on promising results from the Phase 2 MERIT-1 clinical trial (NCT02021292) that…
