News

Apabetalone (RVX-208), Resverlogix‘s experimental treatment for pulmonary arterial hypertension (PAH), was well-tolerated in a small clinical trial, the company announced. The trial met its primary goal of adding apabetalone to standard care to improve pulmonary vascular resistance (PVR), a measure that reflects the internal resistance to blood…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running an endurance race they are highly motivated to complete, but these incremental burdens negatively impact their ability or willingness to…

Breathing oxygen-enriched air can significantly improve exercise performance in people with pulmonary hypertension (PH) linked to heart failure with preserved ejection fraction (HFpEF), including those who might not use supplemental oxygen, according to a small clinical study. Better blood-oxygen levels and more efficient breathing (exchange of carbon dioxide and…

For premature babies who are at high risk of pulmonary hypertension (PH) and other cardiovascular complications, consuming human breast milk in the first year of life is associated with better heart function, a new study found. “Preterm infants have abnormal heart function,” Afif EL-Khuffash, MD, the study’s lead author, said…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both in-person and online. “Supporting charitable causes and organizations are a long-standing tradition of the TCS New York City…

Early treatment with Janssen’s Uptravi (selexipag) can halve the risk of disease progression for people with pulmonary arterial hypertension (PAH), a new analysis found. The results showed patients treated with Uptravi within six months of PAH diagnosis saw their risk of disease progression reduced by 52% compared with…

Higher levels of chloride in pulmonary artery smooth muscle cells (PASMCs) may contribute to the development of pulmonary hypertension (PH) after exposure to low oxygen (hypoxia), a study in a rat model suggested. The study, “Increased intracellular Cl− concentration in pulmonary arterial myocytes is associated with chronic hypoxic…

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…

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…