News

Transition from phosphodiesterase-5 inhibitors (PDE5i) — such as United Therapeutics’ Adcirca (taladafil) and Pfizer‘s Revatio (sildenafil) — to Bayer‘s Adempas (riociguat), a soluble guanylate cyclase (sGC) stimulator, without a washout period is a viable option for some patients with pulmonary hypertension, a small study suggests. The…

Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…

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…