News

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

Treatment with a single medication (monotherapy) may be sufficient to help manage people with “atypical” pulmonary arterial hypertension (PAH), according to an analysis from four clinical centers in Germany. One-year survival of adults diagnosed with “atypical” PAH — those older than 65 and with multiple risk factors for heart…

The U.S. Food and Drug Administration (FDA) has accepted an updated application asking that the dry-powder inhalation therapy LIQ861 (treprostinil) be approved to treat pulmonary arterial hypertension (PAH). Liquidia Corporation, the therapy’s developer, first…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Pregnant women with high pressure in the pulmonary artery of the heart, but who are not considered at risk of pulmonary hypertension (PH), are likely to also be at lower risk of other major cardiac problems, researchers reported. In this study, the level of PH probability was judged based…

Scientists have created a new “cellular blueprint” of healthy lung cells that provides a reference against which tissue from lungs affected by conditions like pulmonary hypertension (PH) can be compared. The ability to accurately map the differences between healthy and diseased lungs can help researchers find new therapeutic approaches,…

Sandoz‘s generic treprostinil injection now is available for subcutaneous (under-the-skin) administration to treat  pulmonary arterial hypertension (PAH), the company announced in a press release. Treprostinil first was approved to treat PAH in the U.S. in 2002. It was developed by United Therapeutics, which markets the…

Trifluoperazine or TFP, an approved schizophrenia medication, also may be effective in the treatment of pulmonary arterial hypertension (PAH), a preclinical study suggests. In rat models of PAH, trifluoperazine was found to provide therapeutic benefits — without causing detectable side effects. The study, “Preclinical Investigation of Trifluoperazine…

A new analysis suggests that the doses of rodatristat ethyl — an investigational therapy for pulmonary arterial hypertension (PAH) — now being evaluated in the Phase 2b ELEVATE 2 clinical trial are likely to reduce levels of the hormone serotonin to an extent that has been demonstrated to…

Keros Therapeutics’ experimental therapy KER-012 prevented increases in the levels of markers for inflammation, fibrosis (scarring), and heart damage in a rat model of pulmonary arterial hypertension (PAH), a study shows. “We believe that the current study supports that KER-012 has the potential to treat PAH in patients without…

Medical care for patients with pulmonary hypertension (PH) was affected during the first wave of the COVID-19 pandemic, with some patients reporting worsening health and difficulties in accessing expert care, a worldwide survey shows. The results were published in the Orphanet Journal of Rare Diseases, in the…

People with pulmonary hypertension (PH) are invited to apply for $500 micro-grants offered by Team PHenomenal Hope to help cover non-medical needs they are struggling to meet due to the COVID-19 pandemic and how it affected them financially. The grants, offered through nonprofit’s Unmet Needs Patient Impact Fund, can be…