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Routine blood screenings and non-invasive clinical tests may predict disease severity and mortality in people with pulmonary hypertension (PH), a study suggested. Specifically, the red blood cell distribution width (RDW) test, used in hematology laboratories to classify anemia, may help in assessing disease severity, while electrocardiographic ratios may predict…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…

Levosimendan, an investigational treatment for pulmonary hypertension and heart failure with preserved ejection fraction, or PH-HFpEF — which showed positive results in a recent clinical trial — may work by reducing the amount of blood putting pressure on the walls of blood vessels, a new analysis suggests. The…

In late July, the U.S. Food and Drug Administration (FDA) approved a new formulation of Janssen‘s Uptravi (selexipag) for use by people with pulmonary arterial hypertension (PAH). Uptravi already was available as an oral tablet. The new formulation is intravenous (IV) — meaning it is administered by an…

A peptide — a short chain of amino acids, the building blocks of proteins — called BPC 157 may prevent or counteract pulmonary arterial hypertension (PAH) and its complications, according to a preclinical study in a rat model of the disease. The findings suggest that BPC 157 may…

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…

Note: This story was updated Aug. 11, 2021, to clarify that the researchers are not affiliated with the Virginia Tech Carilion School of Medicine. Two new researchers at Virginia Tech will be focusing their studies on the mechanisms of heart and vascular diseases — including the role of RNA molecules…

Certain chemokines — signaling molecules that attract white blood cells to sites of infection — may be blood markers of both a person’s risk of idiopathic pulmonary arterial hypertension (PAH) and this disease’s likely course in a patient, a study found. PAH is considered idiopathic when its cause is unknown…

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…

Pulnovo Medical recently raised an undisclosed amount totaling millions of dollars in investment funding to support clinical trials and product development related to pulmonary arterial hypertension (PAH). “The road of global medical innovation has never been an easy journey,” Cynthia Chen, Pulnovo’s executive president, said in a…

The U.S. Food and Drug Administration (FDA) has approved an intravenous form of Uptravi (selexipag) — one infused directly into the bloodstream — for people with pulmonary arterial hypertension (PAH) temporarily unable to take oral medicines. The new intravenous, or IV, formulation is intended to prevent treatment interruptions in adults…

PulmoSIM Therapeutics has joined with scientists at National Jewish Health hospital and Brown University to advance PT001, its investigational and potentially disease-modifying therapy for pulmonary arterial hypertension (PAH), into clinical trials. The partnership follows PT001 being designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status…