AER-901, an inhaled formulation of imatinib being developed as a treatment for pulmonary arterial hypertension (PAH), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The investigational therapy was designed with the goal of reducing the side effects reported among PAH patients using an…
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A six-month at-home rehabilitation program can help improve respiratory muscle strength and physical endurance, while promoting better quality of life, in people with pulmonary arterial hypertension (PAH) That finding from a small clinical trial revealed benefits extending to six months after the end of the program, and suggests this…
The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The Circle of Care Guidebook for Caregivers of Children With Rare and/or Serious Illnesses” was designed…
Cereno Scientific is planning to begin a Phase 2 clinical trial of CS1 — the company’s lead therapy candidate for pulmonary arterial hypertension (PAH) — this September, following clearance from the U.S. Food and Drug Administration (FDA). The upcoming trial will use Abbott‘s CardioMEMS HF System, a…
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…
