News

Novel cell therapy CAP-1002 safe, shows promise in PAH: Trial

In people with pulmonary arterial hypertension (PAH), treatment with the novel cell therapy CAP-1002 was safe and showed encouraging efficacy, according to the results of a Phase 1a/b clinical trial. In particular, exploratory efficacy measures supported the potential of CAP-1002 in improving cardiopulmonary function, including exercise capacity, in PAH…

Early study of inhaled nitric oxide delivery system doses 1st patient

An early feasibility study evaluating Third Pole Therapeutics‘ eNOfit — a miniaturized, portable inhaled nitric oxide (iNO) delivery system to treat pulmonary hypertension (PH) associated with interstitial lung disease — has dosed its first patient, the company announced. The goal for the investigational device is to provide patients…

Benza named director of PH for Mount Sinai Health System

Raymond Benza, MD, has been selected to be director of pulmonary hypertension (PH) for the Mount Sinai Health System, In this newly created position, Benza will seek to advance and standardize care for PH patients and further develop research in the progressive disorder that is associated with high blood…

Survey reveals patients’ preferences for treating PAH

When choosing a treatment regimen, people with pulmonary arterial hypertension (PAH) consider the impact on physical limitations and survival at three years as the most important parameters to consider, according to a study in Germany. Meanwhile, unplanned hospitalizations within three years and short-term side effects were reported as less…

HDL component ApoA-I predicts mortality in PAH: Study

Lower levels of apolipoprotein A-I (ApoA-I), a component of high-density lipoprotein (HDL), or “good cholesterol,” is a significant predictor of mortality in people with pulmonary arterial hypertension (PAH), a study suggests. “Our study identified the significance of ApoA-I as a biomarker for predicting the survival outcome of PAH patients,”…

SPHK2 protein may offer way to reverse pulmonary hypertension

A protein called SPHK2 helps to drive pulmonary hypertension (PH) by changing the epigenetic profiles of cells in blood vessels, a study reports. Epigenetics refers to changes in gene activity that do not change the genetic code itself. As such, study findings imply that blocking SPHK2 or otherwise reversing…

Results from Phase 2b part of inhaled imatinib trial due in June

The Phase 2b portion of the IMPAHCT clinical trial, which is testing multiple doses of the inhaled imatinib formulation AV-101 against a placebo in adults with pulmonary arterial hypertension (PAH), has finished enrollment. Top-line results are expected in June 2024, according to AV-101’s developer, Aerovate Therapeutics. “The completion…

PH treatment aids children having congenital heart disease surgery

Treating children with Revatio (sildenafil) reduced pulmonary arterial pressure after surgery for congenital heart disease (CHD), according to a new analysis. The pulmonary hypertension (PH) treatment, given between one and two weeks before surgery, reduced how long cardiopulmonary bypass — a procedure that takes over the functions of the…


A Conversation With Rare Disease Advocates