The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PT001, an investigational treatment for pulmonary arterial hypertension (PAH). The FDA gives this designation to therapies with the potential to treat rare disorders, defined as those that affect fewer than 200,000 people in the U.S. The designation…
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The U.S. Food and Drug Administration (FDA) has granted priority review status to the application seeking approval of Tyvaso DPI — a dry powder inhaled formulation of treprostinil — for the treatment of both pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Priority review shortens…
Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…
A combination of lung injury and increased activation of B-cells — immune cells responsible for antibody production — was sufficient to promote symptoms of pulmonary hypertension (PH) in mice, a study shows. In addition, increased B-cell activation and higher levels of immune cells known to promote antibody production also were…
Despite a high-risk of serious complications, 10 women with moderate to severe pulmonary arterial hypertension (PAH) had a successful pregnancy and delivery, according to a single center study. The patients were managed with prostacyclin therapy in addition to PAH-specific treatments during pregnancy and specialized care. Most transitioned to oral…
A three-month unsupervised health intervention based on a commercial activity tracker and motivational texts significantly increased daily step counts and improved quality of life in adults with pulmonary arterial hypertension (PAH), according to data from a single-center trial in the U.S. Since physical activity has been shown to…
A new metric for assessing cardiac health, called pulmonary artery proportional pulse pressure or PAPP, could help to identify people at high risk of hospitalization or death due to pulmonary hypertension or heart failure, allowing for more timely and effective treatment, a study reported. Researchers at the University of…
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…
