High pulmonary pressure, cardiac medication use, and poor heart function are all risk factors for pulmonary hypertension (PH) in adults after a procedure to repair an atrial septal defect, a hole in the wall (septum) that divides the two upper chambers of the heart, a study reports. The study,…
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…
Children with idiopathic pulmonary hypertension (IPH) who underwent lung or combined heart-lung transplants show similar survival rates to children who underwent these procedures for other indications, according to a study. Data also indicated that children who had a transplant at an older age had better outcomes. The study, “…
Hello, World! For those of you who don’t know me, my name is Noah Greenspan. I am the founder and program director of the Pulmonary Wellness & Rehabilitation Center in New York City. I am also the founder of the Ultimate Pulmonary Wellness Webinar Series, the Ultimate…
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…
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