People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…
Switching from the approved Tyvaso (inhaled treprostinil) nebulizer to Tyvaso DPI — an investigational dry-powder formulation — is safe, effective, and likely more convenient for people with pulmonary arterial hypertension (PAH), top-line data from the Phase 1 BREEZE trial show. These positive findings also were supported by data from…
Researchers have identified a new mutation in the FLNA gene that was linked with pulmonary arterial hypertension (PAH) and congenital heart defects in a two-year-old girl. This case, along with more than a dozen others, highlights the association between FLNA mutations and early onset PAH. Together, they support genetic testing…
Long-term treatment with Bayer’s Adempas (riociguat) was safe and well-tolerated in people with pulmonary arterial hypertension (PAH), according to data from the real-world EXPERT study. These findings from clinical practice were consistent with those reported in previous clinical trials, supporting the therapy’s favorable safety profile. Data from EXPERT…
The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
Acceleron Pharma announced plans to launch the first Phase 3 clinical trial, called STELLAR, evaluating the safety and effectiveness of its investigational therapy sotatercept (ACE-011) in people with pulmonary arterial hypertension (PAH), before the end of the year. Two other Phase 3 trials testing sotatercept as an…
Suppressing CD248, a cell surface protein, reduced pulmonary vascular remodeling — the key structural alteration in pulmonary arterial hypertension (PAH) — in a rat model of the disease. CD248 was found to promote PAH-associated abnormal growth and migration of pulmonary artery smooth muscle cells (PASMCs), the cells that line the…
Mallinckrodt Pharmaceuticals’ INOmax (nitric oxide) gas improves oxygenation in premature newborns with pulmonary hypertension (PH) at least as effectively as it does  in term and near-term babies, data from a Phase 4 observational study show. These findings, from a second planned interim study analysis (at 75% enrollment),…
People with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) — particularly those showing low or intermediate disease risk, typical PAH — benefit most from combination therapy with Letairis (ambrisentan) and Adcirca (tadalafil) than from either therapy alone, according to an exploratory sub-analysis of a Phase 3…
Oral delivery of two proteins produced on lettuce leaves and involved in blood vessel health eased pulmonary hypertension and heart abnormalities in a rat model of pulmonary arterial hypertension (PAH). These results point to this plant-based approach as a potentially safe, inexpensive, shelf-stable treatment for PAH. Further studies…
Blood levels of uric acid may be a predictive biomarker of pulmonary arterial hypertension (PAH) in people with scleroderma, according to a recent study. The levels of this normal body waste product, formed when chemicals called purines break down, also may be a biomarker for disease…
Riociguat increases the risk of death and serious adverse events in people with pulmonary hypertension (PH) associated with idiopathic interstitial pneumonia (IIP), and should not be prescribed to those patients, final results of a Phase 2 study show. The study, “Riociguat for idiopathic interstitial pneumonia-associated pulmonary hypertension…
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