News

More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated for COVID-19 if a vaccine was approved under emergency use authorization instead of the routine process, the EveryLife Foundation for Rare Disease reports. These findings…

United Therapeutics has submitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Tyvaso DPI, a dry powder inhaled formulation of treprostinil, for both pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). To expedite the process, the company  applied…

Pulmonary hypertension (PH) increases the likelihood of a prolonged hospitalization following an acute ischemic stroke, or a stroke due to a blocked vessel carrying blood to a part of the brain, according to a large U.S. database study. Men with PH and such a stroke are also 31% more…

Treatment with a fungus called Ophiocordyceps sinensis eased disease signs and improved survival in a rat model of pulmonary arterial hypertension (PAH), suggesting a potential to treat patients. The findings were in the study “Substantial involvement of TRPM7 inhibition in the therapeutic effect of Ophiocordyceps sinensis on…

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a second year, the #RAREis Scholarship Fund — supported by Horizon Therapeutics – will award 35 one-time scholarships, each…

Treatment with the investigational medication levosimendan was found to improve exercise tolerance, and some parameters related to heart function, in a small clinical trial of people with pulmonary hypertension and heart failure with preserved ejection fraction — a condition known as PH-HFpEF. In fact, 84% of patients in the…

More than three-quarters (76%) of adults with pulmonary arterial hypertension (PAH) being treated with Uptravi (selexipag) either saw no increase or a decline in their risk of death over one year, a study based real-world data reports. These findings — in nearly 500 people taking part in an…

Patients whose pulmonary hypertension is associated with chronic obstructive pulmonary disease (PH-COPD) have a worse clinical status and a poorer outcome than those with idiopathic pulmonary arterial hypertension (IPAH), a study based on real-world data from the COMPERA registry reports. Although PH is typically mild to moderate in COPD,…

Treatment with Acceleron Pharma’s sotatercept appears to be effective and generally well tolerated in people with pulmonary arterial hypertension (PAH), according to new data from the ongoing PULSAR and SPECTRA Phase 2 clinical trials. These data will be presented at the American Thoracic Society (ATS) 2021 International…

Diminazene aceturate (DIZE), an approved antiparasitic medication, lessened the development of pulmonary arterial hypertension (PAH) in a rat model, a study has demonstrated. These findings support further investigation of DIZE as a potential treatment for PAH, scientists said.  The…

A ketogenic diet — one that’s low in carbohydrates and higher in fat  — given under medical supervision seems to be safe and effective in treating pulmonary hypertension (PH) when it is associated metabolic syndrome, a case report suggests. The report, “Nutritional ketosis to treat pulmonary…

Tyvaso (inhaled treprostinil) has become the first treatment approved in the U.S. for people with pulmonary hypertension associated with interstitial lung disease (PH-ILD). The U.S. Food and Drug Administration (FDA) approved Tyvaso, developed by United Therapeutics, to improve exercise ability in PH-ILD patients, the company said. “The…