News

The likelihood of developing pulmonary hypertension (PH) is low among people in southern Italy, suggests a study that draws from a random and large grouping of individuals with echocardiograms, done to determine disease prevalence. Independent predictors of PH include female sex and right ventricular enlargement, its scientists noted. The study,…

People with stable pulmonary arterial hypertension (PAH) can safely switch from Tracleer (bosentan) to Letairis (ambrisentan) without compromising their heart function or general health status, a study from China reports. PAH patients in Hunan province may also benefit financially by moving from Tracleer to Letairis, as it…

At its first virtual investor event, biotech company Centogene set a bold mission: to cure 100 rare diseases within the next decade. A leader in the field of genetic diagnostics, Centogene used the June 22 event to present its strategic priorities, outlining its plans to speed the discovery…

Joining support groups — meeting people with common experiences and concerns who can provide emotional reassurance — for pulmonary hypertension (PH) helped patients manage symptoms, stick to medicines, and better understand their disease, a study reports. Participation in a support group, however, had no impact on the general quality…

A study of rare gene variants has identified two new candidates — fibulin 2 (FBLN2) and platelet-derived growth factor D (PDGFD) — that increase risk for adult-onset idiopathic pulmonary arterial hypertension (IPAH). In-depth genetic analyses also predicted that about 15% of IPAH cases in children are caused by de…

Dosing has begun in a clinical trial testing AER-901, Aerami Therapeutics‘ inhaled formulation of imatinib, which is being investigated as a potential treatment for pulmonary arterial hypertension (PAH). “We are pleased to announce that the dosing of subjects in our Phase 1 trial is ongoing and that we expect to…

The Center for Innovative Technology (CIT) GAP Funds has invested in OxiWear and its ear-wearable device designed to continuously monitor blood oxygen levels in people with pulmonary hypertension (PH) and alert them when levels fall below safe thresholds. Oxiwear is expected to help patients get the help they…

To help patient advocacy leaders and their partners better understand how global health statistics codes — known as ICD codes — are assigned, updated, and revised in the U.S. health information system, the EveryLife Foundation for Rare Diseases is presenting a first-of-its-kind resource guide. The foundation created the…

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…

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…