Yutrepia, an inhaled formulation of treprostinil, was generally well tolerated and improved the quality of life for people with pulmonary arterial hypertension (PAH) in the Phase 3 INSPIRE clinical trial. The therapy was preferred by nearly all trial participants who switched to it from Tyvaso, an older inhaled…
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When done during pregnancy, an echocardiogram — a noninvasive measurement of heart function that uses sound waves — may help doctors predict pulmonary hypertension in newborns, a study in China suggests. An echocardiogram can show how well the heart’s right ventricle, one of its bottom pumping chambers, will push…
More than half of people with pulmonary arterial hypertension (PAH) who start daily treatment with oral Opsumit (macitentan) are expected to still be alive after nine years on the therapy, according to a new analysis of data from the SERAPHIN clinical trial and its open-label extension. “These analyses…
Treatment with Uptravi (selexipag) significantly improved blood flow parameters in people with inoperable or persistent chronic thromboembolic pulmonary hypertension (CTEPH) in a Phase 3 clinical trial, although its use did not significantly affect measures of physical abilities. Full trial results are in “Selexipag for the treatment of…
Among people with chronic thromboembolic pulmonary hypertension (CTEPH), risk assessments and treatment patterns prior to surgical procedures do not predict outcomes after surgery, a new study from Sweden shows. The results suggest that most CTEPH patients who don’t undergo surgery remain in the same risk category for at least…
A gene therapy designed to provide a protein implicated in heart function improvement was found to lessen pulmonary blood vessel remodeling and reduce lung blood pressure in a preclinical study of pulmonary arterial hypertension (PAH). The study, “Apela gene therapy alleviates pulmonary hypertension in rats,” was published in…
Pulnovo Medical reported that it “successfully concluded” a first meeting with the U.S. Food and Drug Administration (FDA) regarding the design of a pivotal clinical trial of its pulmonary artery denervation (PADN) device in people with pulmonary arterial hypertension (PAH). PADN aims to lower pulmonary artery pressure by…
Higher levels of the metal antimony were found in the bloodstream of adults with pulmonary arterial hypertension (PAH) than those without the condition, a pilot study reported. Elevated antimony levels also correlated with higher blood pressure in the pulmonary arteries and reduced heart function. The pilot study, “Plasma…
When Zac Gunn was diagnosed with pulmonary hypertension (PH) three years ago, he was in very poor shape. But he rallied, astounding his medical team with his resilience and zest for life. And recently, the 4-year-old, who is awaiting a lung transplant, went on stage to receive the Child…
People with pulmonary arterial hypertension (PAH) have a distinct immune profile compared with healthy individuals, according to a new study that also found different activity levels in PAH patients of genes involved in pulmonary vascular remodeling, oxygen transport, and blood cell development. In fact, more than 1,200 genes were…
Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.
The use of the hormone vasopressin successfully stabilized symptoms associated with pulmonary hypertension (PH) in newborns, and avoided the need for more invasive treatment approaches in some infants, according to data from 10 cases. “Although our findings are exploratory and further research is needed to establish safety and efficacy,…
