RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their own disease registries. The site was developed by the National Center for Advancing Translational Sciences (NCATS), a division of the National Institutes of…
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A type of abnormality in small blood vessels of the eyes, called retinal vascular tortuosity, may provide a non-invasive way to detect alterations in lung circulation in patients with pulmonary arterial hypertension (PAH), according to a new study. The research, “Retinal Vascular Tortuosity, a New Feature…
A new analysis of Actelion‘s GRIPHON clinical trial indicates that Uptravi (selexipag) tablets reduce the risk of disease complications or death in patients with pulmonary arterial hypertension (PAH), regardless of when treatment begins. However, its effectiveness was greater if started sooner after diagnosis. The data was presented at…
Balloon pulmonary angioplasty reduces sleep apnea in chronic thromboembolic pulmonary hypertension (CTEPH) patients, according to a study. The observed improvements further correlated with a better mean pulmonary arterial pressure. The results from the study, “Balloon pulmonary angioplasty attenuates sleep apnea in patients with chronic thromboembolic pulmonary hypertension,” was…
Promising preclinical findings recently published in a peer-reviewed journal opened the door to the launch of a Phase 2 trial testing the therapeutic potential of apabetalone (RVX-208), Resverlogix‘s proprietary therapy, in people with pulmonary arterial hypertension (PAH). The findings of the study, “Multicenter Preclinical Validation of…
With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies(NCATS) at the…
Bellerophon released an update on the latest data regarding its INOpulse therapy, and announced its plans to apply for the therapy’s approval as a treatment for patients with pulmonary hypertension associated with interstitial lung disease (PH-ILD). INOpulse is a device that uses inhaled nitric oxide (iNO),…
Survival rates of patients with chronic thromboembolic pulmonary hypertension (CTEPH) unable to undergo surgical treatment have significantly improved in recent years, a retrospective study has found. This suggests that minimally invasive balloon pulmonary angioplasty and other interventions implemented more recently can improve CTEPH patients’ prognoses. The study, “Predictors…
INOmax (nitric oxide) gas improved the oxygenation of premature, term, and near-term newborns with pulmonary hypertension (PH) and associated hypoxic respiratory failure, according to data from a interim analysis of Mallinckrodt Pharmaceuticals’ Phase 4, observational PaTTerN registry study. Persistent PH in newborns occurs when the pressure in…
A natural component of milk thistle seeds called silibinin can lessen some early signs of pulmonary arterial hypertension (PAH) in rats, likely by inhibiting a cellular signaling pathway suspected to contribute to disease progression, a pilot…
Teva Pharmaceutical Industries launched a generic version of Letairis (ambrisentan) in the United States for the treatment of pulmonary arterial hypertension. Generic ambrisentan will be available as 5 and 10 mg tablets. Letairis is marketed by Gilead in the U.S., while in Europe it is sold…
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how much harder it is for patients with rare diseases who must get to doctors’ appointments or clinical trials that are hundreds of miles away from home.
