News

The European Commission has granted orphan drug designation to sotatercept for the treatment of people with pulmonary arterial hypertension (PAH). This designation is given to medications with the potential to substantially improve care for rare diseases — defined as those that affect fewer than five individuals per 10,000…

Treatment with bosentan may lower the risk of developing pulmonary hypertension (PH) and help stabilize lung function in scleroderma patients with digital ulcers, a study suggests. Patients who never had taken bosentan had a 3.9 times increased risk for PH compared to those on this therapy, findings…

Lower quality of life in patients with pulmonary hypertension (PH) was found to be associated with higher burden experienced by their caregivers, according to a study conducted in Turkey. The study, “Caregiver Burden in Patients with Pulmonary Hypertension,” was published in the journal Clinical Nursing…

People with both pulmonary hypertension (PH) and interstitial lung disease (ILD) have worse survival outcomes than ILD patients who don’t have PH, a study has found.  Lung function and blood flow tests were important predictors of overall survival in this patient population, according to the study, “…

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 patient has enrolled in REBUILD, a Phase 3 clinical trial that will assess the safety and efficacy of Bellerophon Therapeutics’ INOpulse, an experimental treatment for patients with pulmonary fibrosis (PF) who are at risk of developing pulmonary hypertension…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…

The Pulmonary Hypertension Association U.K. (PHA UK), a charity organization dedicated to supporting those with pulmonary hypertension (PH), is urging adults with PH to participate in a research study assessing the usefulness of a self-help program to manage anxiety. By participating in the study patients will have…

The U.S. Food and Drug Administration (FDA) determined that additional data is required to support the potential approval of LIQ861, Liquidia Technologies’ investigational inhaled formulation of treprostinil, for the treatment of pulmonary arterial hypertension (PAH). The U.S. agency issued a complete response…

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…

Liquidia Technologies, the developer of an inhaled formulation of treprostinil (LIQ861) for the treatment of pulmonary arterial hypertension (PAH), has completed its acquisition of RareGen, the marketer of generic treprostinil injections. The…