News

A group of Canadian doctors highlighted the importance of an early diagnosis among those with rare diseases. The recommendation, released by the Pulmonary Hypertension Association of Canada, came out on Rare Disease Day, an international event every Feb. 28 to raise awareness of the 300 million people with rare…

Compound 3 is a potent and selective inhibitor of ROCK-1 and ROCK-2 — proteins associated with blood vessel contraction — and can ameliorate disease features of pulmonary hypertension (PH), a study finds. The study “Pharmacological characterization of a highly selective Rho kinase (ROCK) inhibitor and its therapeutic effects in experimental…

The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…

Treatment with Revatio (sildenafil) after surgery to the heart’s mitral valve significantly lowered pulmonary arterial pressure in patients with pre-operative significant pulmonary hypertension, researchers from Israel report. The study, “Sildenafil for Pulmonary Hypertension in the Early Postoperative Period After Mitral Valve Surgery,” was published in the Journal…

The first patient has been given the investigational therapy CXA-10 in an ongoing Phase 2 trial in patients with pulmonary arterial hypertension (PAH), Complexa, the product’s developer, announced. The PRIMEx study (NCT03449524) was planned to evaluate the safety, efficacy, and overall stability and distribution in the…

Patients with chronic thromboembolic pulmonary hypertension (CTEPH) and pulmonary arterial hypertension (PAH) have higher serum levels of antibodies against the EDX2 and PHAX proteins, according to research which suggests that these autoantibodies could be used as diagnostic tools for both diseases. The study, “Elevated levels…

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…

Greater experience in balloon pulmonary angioplasty (BPA) enables shorter hospital stays for patients with chronic thromboembolic pulmonary hypertension (CTEPH), reduces use of intensive care, and lowers medical costs while maintaining the procedure’s safety, according to new data from real-world practice in Japan. The study, “Shortening Hospital Stay Is…

Dacomitinib, a medicine recently approved by the U.S. Food and Drug Administration for the treatment of certain forms of lung cancer, showed promising therapeutic effects in two animal models of pulmonary arterial hypertension (PAH), a study reports. The study, “Dacomitinib, a New Pan-EGFR Inhibitor, is Effective in…

Invasive pulmonary artery denervation is safe and can improve the outcome of patients with combined pre- and post-capillary pulmonary hypertension associated with left heart failure, compared with standard of care, results of a randomized trial show. The findings were reported in the study “Pulmonary Artery Denervation…

Acute treatment with Ventavis (iloprost) improves the heart’s right ventricular (RV) function in patients with idiopathic pulmonary arterial hypertension (IPAH) and PAH associated with connective tissue disease (CTD-PAH), according to a study. The study, “Acute Iloprost Inhalation Improves Right Ventricle Function in Pulmonary Artery…

An updated guideline with new recommendations for the treatment of patients with pulmonary arterial hypertension (PAH) was released by the American College of Chest Physicians (CHEST). The free guide, titled “Therapy for Pulmonary Arterial Hypertension in Adults 2018: Update of the CHEST Guideline and Expert Panel Report…