News

MACC1 seen as biomarker for PAH in bioinformatics study

MACC1, a gene that’s been linked to overgrowth of cancer cells, is more active in the lungs of people with pulmonary arterial hypertension (PAH) than in healthy individuals, a study out of China found, suggesting it could be used as a diagnostic biomarker for the disease. Researchers also observed…

Low circALMS1 levels could be prognostic biomarker in PH: Study

Low levels of circular RNA Alstrom syndrome protein 1, or circALMS1, may be associated with poor outcomes with pulmonary hypertension (PH), a study suggests. This may be because low circALMS1 levels promote higher numbers and the migration of pulmonary microvascular endothelial cells (PMECs), and reduce the cells’ death. PMECs…

Opsumit shows real-world safety, efficacy for CTD-PAH patients

Most people with pulmonary arterial hypertension (PAH) due to connective tissue disease (CTD) who are using Opsumit (macitenan) do so as part of a combination therapy, according to real-world evidence from two U.S. drug registry studies. Its safety and effectiveness, in terms of clinical outcomes, for these patients…

PH raises risk of severe cardiac, respiratory issues in HIV patients

Having pulmonary hypertension (PH) worsens the likelihood of potentially life-threatening cardiac and respiratory complications in people with human immunodeficiency virus (HIV) infection, a U.S. database study reported. Its findings “can inform clinical practice and help healthcare providers make more informed decisions regarding the screening, diagnosis, and management of PH…

Trial enrollment begins to test long-acting relaxin formula in PH-HFpEF

Enrollment has begun in a Phase 1b study of Tectonic Therapeutic’s TX45, an investigational treatment for people with pulmonary hypertension and heart failure with preserved ejection fraction (PH-HFpEF). TX45 is a long-acting formulation of relaxin, a protein involved in reproduction and cardiovascular function. It has anti-fibrotic, or anti-scarring,…

FDA approves sotatercept, now Winrevair, to treat PAH in adults

The U.S. Food and Drug Administration (FDA) has approved the under-the-skin injection therapy sotatercept-csrk — to be marketed under the brand name Winrevair — to treat adults with pulmonary arterial hypertension (PAH). The now-approved treatment is expected to be available for dispensing by select U.S. specialty pharmacies by the…


A Conversation With Rare Disease Advocates