The Black Women’s Health Imperative (BWHI) recently created a Rare Disease Diversity Coalition focused on reducing racial disparities in the rare disease community. Getting a timely and accurate diagnosis for a disease that few people — sometimes even physicians — have heard of is challenging on its own merit.
People with pulmonary arterial hypertension (PAH) who were treated with Uptravi (selexipag) had a lower risk and rate of hospitalization than those taking Orenitram (treprostinil), a real-world analysis revealed. The study,…
Six weeks of treatment with levosimendan significantly lowered pressure on the heart and improved physical capacity among people with pulmonary hypertension and heart failure with preserved ejection fraction (PH-HFpEF), findings from a Phase 2 trial show. The investigational treatment, by Tenax Therapeutic, however, did…
Bayer has opened its second annual Pulmonary Hypertension Accelerated Bayer (PHAB) Awards, a grant funding program to support clinical research projects into pulmonary hypertension (PH), particularly those focused on pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). The program, one of…
A composite assessment of voice features may be able to monitor changes in the pulmonary arteries of people with pulmonary hypertension (PH) who are at risk of heart failure, helping identify those more likely to experience poor outcomes, a study found. While these patients usually require invasive tests to…
Pulmonary artery denervation was found to be safe and to improve the health of people with pulmonary arterial hypertension (PAH), according to results of an early clinical trial. The study, “Intravascular Ultrasound Pulmonary Artery Denervation to Treat Pulmonary Arterial Hypertension (TROPHY1),” was published in the journal …
Changes in the epigenetic profile — a collection of marks that turn genes on and off  — of pulmonary arterial endothelial cells from people with pulmonary arterial hypertension (PAH) may contribute to disease development, a study suggests. The study, “Remodeling of active endothelial enhancers is associated with aberrant…
While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…
Kalytera Therapeutics intends to acquire Salzman Group, a company developing R-107 — a liquid form of nitric oxide — to possibly treat pulmonary arterial hypertension (PAH), lung disease associated with COVID-19, and other lung disorders, the company announced. Its acquisition of the Massachusetts-based company should be completed by…
European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Chronic inhalation of nicotine, the addictive component of all tobacco products, is enough to trigger changes in pulmonary arteries and in the heart’s right chamber that can cause pulmonary hypertension (PH), a recent study in mice found. The study, “Effects of Chronic Nicotine Inhalation on…
Newly published research has detailed the underlying biochemical process behind Acceleron Pharma’s investigational therapy sotatercept (ACE-011) for the potential treatment of people with pulmonary arterial hypertension (PAH). The findings, in two rat models of PAH,…
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