The first participant has been enrolled in a Phase 2 trial testing the investigational therapy levosimendan in pulmonary hypertension (PH) patients with heart failure and preserved ejection fraction (PH-HFpEF). Ejection fraction refers to how much blood the heart’s left ventricle pumps out with each contraction. In PH-HFpEF, the…
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…
Women’s pulmonary artery muscle cells produce more of a signal molecule called hypoxia-inducible factor 1-alpha (HIF1α), which may contribute to the higher prevalence of pulmonary arterial hypertension (PAH) in women compared to men, a study reports. The data also suggest that a component of…
Spouses of people with pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH) report feeling insecure about their situation and dissatisfied with the degree of information and support provided by healthcare teams, according to…
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…
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