Note: This story was updated Sept. 28, 2020, to clarify that Uptravi was being tested as part of an initial triple combination strategy with Opsumit and tadalafil for newly diagnosed patients. An initial triple combination strategy of the oral therapies Uptravi (selexipag), Opsumit (macitentan),…
Using sildenafil and milrinone together proved more effective than either therapy alone in treating newborns with severe persistent pulmonary hypertension (PH), a recent clinical study indicates. This combo treatment may be used in resource-constrained settings that lack inhaled nitric oxide or extracorporeal membrane oxygenation (ECMO) as…
Adults with chronic thromboembolic pulmonary hypertension (CTEPH) experience more breathing stoppages during sleep (apnea), lower blood oxygen, and higher levels of pro-inflammatory proteins in the blood than people with idiopathic pulmonary arterial hypertension who…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
Despite current targeted therapies, premature babies with bronchopulmonary dysplasia or BPD — a disease in which the lungs become irritated and fail to develop properly, causing breathing difficulties — associated with pulmonary hypertension (PH) continue to have high mortality rates, a study found. Nevertheless, infants who live past…
Liquidia Technologies has received a notice of allowance from the U.S. Patent and Trademark Office, indicating that a patent application for LIQ861, the company’s investigational dry powder formulation of treprostinil, should be approved. The patent is expected to cover methods of treating patients with…
Robot-assisted pulmonary artery denervation (PADN) — a minimally invasive surgical procedure that destroys some of the nerves innervating the pulmonary artery to reduce blood pressure — can lower heart strain and improve exercise capacity in patients with chronic thromboembolic pulmonary hypertension (CTEPH). That finding was reported in…
Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
High levels of the peptide adiponectin show potential as a biomarker for the prognosis of congenital heart disease in patients with pulmonary hypertension (PH), according to a recent study. Titled “High plasma adiponectin is associated with increased pulmonary blood flow and reduced right…
Endovascular embolization — a minimally invasive surgery that cuts off blood flow from a specific group of blood vessels in the body — can increase the chances of survival in babies with vein of Galen aneurysmal malformation (VGAM) and severe pulmonary hypertension (PH), a study reports. Findings from…
The U.S. Food and Drug Administration (FDA) has accepted for review the  application for Tyvaso (inhaled treprostinil) as a potential treatment for pulmonary hypertension associated with interstitial lung disease (PH-ILD). The supplemental new drug application (sNDA) was submitted by United Therapeutics,…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
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