News

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…

Adverse effects are associated with poor adherence to phosphodiesterase-5 inhibitors (PDE-5I) as treatment for patients with pulmonary arterial hypertension (PAH) treated via an integrated care model at an academic institution. Hospitalization events, out-of-pocket costs and frequency of dosing did not play a role, a study shows. Led by researchers at…

A new generic version of Actelion’s Tracleer (bosentan) developed by Teva Pharmaceutical was approved by the U.S. Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH). The decision was supported by evidence that the generic bosentan has similar biochemical properties to…

Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…

A new meta-analysis assessing balloon pulmonary angioplasty (BPA) and Adempas (riociguat) for treating inoperable chronic thromboembolic pulmonary hypertension (CTEPH) found both treatment strategies to be well-tolerated, and able to improve exercise tolerance and the function of the circulatory system. The study with that finding, “Balloon…

Note: This is the second article in a three-part series written in collaboration with respiratory therapist Mark W. Mangus Sr., RRT, RPFT, FAARC, and oxygen expert Ryan Diesem. The first part can be found here. When it comes to home improvement, people typically want three things from a contractor:…

Patients with idiopathic pulmonary arterial hypertension (IPAH) have greater levels and activity of a chloride channel known as TMEM16A, which contributes to disease-related changes in pulmonary cells, according to a new study. The findings also revealed that long-term treatment with benzbromarone, a TMEM16A blocker, induces relaxation of blood…

People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most lawmakers aren’t experts in even one well-known disease — let alone the world’s estimated 7,000 rare disorders. So how does the…

Treatment for 12 weeks with Actelion Pharmaceuticals’ Opsumit (macitentan) significantly improved blood vessels’ resistance in patients with portopulmonary hypertension without causing further damage to the liver, data from the PORTICO Phase 4 trial show. The study, “Macitentan for the treatment of portopulmonary hypertension (PORTICO): a multicentre, randomised, double-blind,…

A new study in mice demonstrated that a lack of iron in muscle cells lining the circulatory system in the lungs — termed pulmonary arterial smooth muscle cells (PASMCs) — sets off a chain of events that result in pulmonary hypertension (PH). These results suggest a cause-and-effect link between iron…

Exposure to air pollutants may be linked to disease severity and other clinical outcomes in people with pulmonary arterial hypertension (PAH), a study shows. The study, “Traffic exposures, air pollution and outcomes in pulmonary arterial hypertension: a UK cohort study analysis,” was published in the European Respiratory…

The U.S. Food and Drug Administration (FDA) has approved Novitium Pharma‘s generic sildenafil for oral suspension at a dose of 10 mg/mL for the treatment of pulmonary arterial hypertension (PAH). This product, which had been designated a competitive generic therapy by the FDA, is the first…