Forest Ray PhD,  —

A first patient has been dosed in a Phase 1b trial evaluating the safety and efficacy of GMA301, an investigational antibody for the treatment of pulmonary arterial hypertension (PAH), Gmax Biopharm, the therapy’s developer, announced in a press release. GMA301 is a humanized monoclonal antibody,…

Janssen Pharmaceutical has asked the U.S. Food and Drug Administration (FDA) to approve the use of intravenous Uptravi (selexipag) by pulmonary arterial hypertension (PAH) patients unable for a given time to use Uptravi as a prescribed oral treatment. The intravenous, or IV (into-the-vein), formulation is meant to prevent treatment interruptions…

Rare variants of the ABCC8 gene were found among pulmonary arterial hypertension (PAH) patients in Spain, a study reports. The variants, or mutations, are predicted to alter the SUR1 protein that the ABCC8 gene provides instructions to make, but exactly how these variants might affect PAH remains unknown.

Delayed diagnosis, limited resources, and a lack of awareness compound barriers to care for people with pulmonary hypertension (PH) in low- and middle-income countries, a recent review found. The finding highlights the need for better patient advocacy across all levels of care, more affordable diagnostic tools, and improved awareness,…

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…

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…

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…

Aerovate Therapeutics received $72.6 million in Series A funding to advance trials testing AV-101, an experimental inhaled version of imatinib for the treatment of pulmonary arterial hypertension (PAH). AV-101 is an inhaled dry powder that delivers imatinib directly to a patient’s lungs.

Pregnant women with pulmonary arterial hypertension (PAH) should seek specialist care before giving birth and for months afterward, due to the risk of severe maternal complications and high rates of premature births, according to a study in China. The study, “Pregnancy outcomes in patients with pulmonary…

A protein called interleukin-32 may serve as a biomarker for pulmonary arterial hypertension (PAH) in scleroderma patients, for whom PAH is a severe complication. The study reporting the findings, “Interleukin-32 in systemic sclerosis, a potential new biomarker for pulmonary arterial hypertension,” was published in the journal …