News

American researchers have identified three blood-flow measurements that could help doctors predict the outcomes of people whose pulmonary hypertension stems from a serious heart condition. The discovery applies to PH patients whose lung blood pressure disorder is associated with heart failure with preserved ejection fraction, or PH-HFpEF. A diagnosis of…

Deficiency of an enzyme called granzyme B leads to deposits of calcium in pulmonary blood vessels, contributing to the defects found in hypoxic pulmonary arterial hypertension (PAH), an animal study suggests. The study, “Granzyme B deficiency promotes osteoblastic differentiation and calcification of vascular smooth muscle cells in…

Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare diseases currently known — was the focus of a discussion by three top New York geneticists. The Feb. 28 conference,…

Bellerophon Therapeutics is advancing the clinical trials program of its inhaled INOpulse nitric oxide therapy for pulmonary hypertension (PH) associated with several lung diseases. The company is developing INOpulse for patients with pulmonary arterial hypertension (PAH), PH associated with chronic obstructive pulmonary disease (COPD), and PH linked to interstitial lung…

Cardiome recently shared an update on the development of its therapeutic portfolio, including Trevyent (treprostinil injection) for the treatment of pulmonary arterial hypertension (PAH). Following the U.S. Food and Drug Administration’s (FDA) refusal last year to approve a new drug application (NDA) submitted by Trevyent’s licensor SteadyMed,…

When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress — SMARD,  for short. Hunter Pageau, a 12-year-old boy from North Haven, Connecticut, is one of only 80 people in the world known to have SMARD, a motor neuron disease…

Combinations of Reviva Pharmaceuticals’ RP5063 and other pulmonary arterial hypertension treatments improved rats’  lung blood vessel structure and lung function, a study reports. Previous research in rats indicated that RP5063 could improve these measures as a stand-alone therapy. The latest study showed that it worked even better as a combo therapy.

Computer software combined with artificial intelligence can generate advanced imaging techniques for serious diseases such as pulmonary hypertension (PH), improving healthcare, British scientists say. This was the focus of a workshop on digital radiology at Imperial College Academic Health Science Centre (AHSC) that brought together medical researchers, computer scientists, mathematicians…

In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

Levosimendan improved right ventricular function in a rat model of pulmonary hypertension, supporting its potential as an effective therapy for the disease, a study shows. The study, “Levosimendan improves cardiac function and myocardial efficiency in rats with right ventricular failure,” was published in the journal Pulmonary…

MannKind Corporation will start enrollment for a Phase 1 trial to test its treprostinil inhaled therapy for the treatment of pulmonary arterial hypertension (PAH). The trial will be conducted under an Investigational New Drug (IND) application filed with the U.S. Food and Drug Administration (FDA). The company’s…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…