News

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…

A combination of measures of clinical worsening may be meaningful in determining response to therapies in pediatric clinical trials, and a good gauge prognosis in children with pulmonary arterial hypertension (PAH), a registry-based study reports. Disease worsening defined by the occurrence or progression of symptoms, particularly, was found to…

The latest INSPIRE Phase 3 trial data on LIQ861 as a treatment for pulmonary arterial hypertension (PAH) support the submission of a New Drug Application (NDA) with the U.S. Food and Drug Administration. Liquidia Technologies — LIQ861’s manufacturer — announced that it expects to request a pre-NDA…

AIT Therapeutics has obtained a funding agreement that will raise $8 million through private investment in public equity (PIPE) financing to further development of its nitric oxide-based treatments for persistent pulmonary hypertension of the newborn, bronchiolitis, and nontuberculous mycobacteria. Nitric oxide (NO) is a strong vasodilator that opens blood vessels, and is…

Treatment with Revatio (sildenafil) successfully reduced symptoms of portopulmonary hypertension (PPHTN), allowing patients to undergo liver transplant safely, a case report study shows. The case report study, “Sildenafil Monotherapy to Treat Portopulmonary Hypertension Before Liver Transplant,” was published in the journal Transplantation Proceedings. Pulmonary hypertension (PH)…

AIT Therapeutics’ on-demand nitric oxide (NO) ventilator is safer for patients with pulmonary hypertension (PH), and for healthcare workers, compared with standard NO cylinders, a study shows. The study’s results were highlighted in a poster titled “On-Demand Nitric Oxide for Ventilator-Based Nitric Oxide Inhalation: A Risk-Reduction…

New data support the safety and effectiveness of TIVUS (therapeutic intra-vascular ultrasound) for patients with pulmonary arterial hypertension (PAH) on a stable treatment regimen. The data were recently presented at the annual meeting of the European Association of Percutaneous Cardiovascular Interventions (EuroPCR), in Paris. In PAH, small arteries inside…

Owlstone Medical has established a strategic collaboration with Actelion Pharmaceuticals to develop and validate a breath-based diagnostic test for different types of pulmonary hypertension (PH). Actelion Pharmaceuticals will solely fund this discovery and development program. The test will be based on Owlstone’s non-invasive method called Breath Biopsy, in…