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The Pulmonary Hypertension Association of Canada has launched its Life in Purple Challenge campaign to mark November, Pulmonary Hypertension Awareness Month. PH is a rare, progressive and life-threatening disease caused by an increase of blood pressure in the pulmonary arteries and veins, which become narrowed and blocked. Common PH symptoms…

Development work on INOpulse as a treatment for pulmonary hypertension is advancing in all three patient groups, Bellerophon Therapeutics announced when sharing its third quarter financial results. Bellerophon is developing INOpulse — inhaled nitric oxide delivered with the help of a portable device — for patients with pulmonary arterial hypertension…

A generic molecule that works by activating a mitochrondrial protein, dichloroacetate (DCA), was seen in a Phase 1 clinical trial to  reduce pulmonary arterial blood pressure and improve lung function in patients with pulmonary arterial hypertension (PAH). In PAH, cells of blood vessels adopt characteristics that are similar to cancer cells,…

PhaseBio Pharmaceuticals has enrolled and dosed its first patient with pulmonary arterial hypertension (PAH) in a Phase 1 clinical study assessing the safety, tolerability and hemodynamic response to its PAH drug candidate PB1046. Participants are all adult PAH patients with a permanently implanted hemodynamic monitor (CardioMEMS HF System). PB1046,…

Debbie Drell, director of membership at the National Organization for Rare Disorders (NORD), knows all too well about rare diseases. Her older sister, Alex Flipse, was diagnosed with pulmonary hypertension (PH) in September 1998 after enduring five years of worsening symptoms. Drell was told she also had a rare form…

Children’s National Health System no longer treats just kids. Its Rare Disease Institute, launched in April 2017, has partnered with the National Organization for Rare Disorders (NORD) to become the first of many U.S. “centers of excellence” to look after patients with rare diseases, regardless of age. The effort…

A compound that scientists have been examining as a cancer therapy reduced blood pressure in pulmonary hypertension patients’ lungs, according to a clinical trial. Researchers tested the therapy, dichloroacetic acid, in 20 patients with pulmonary arterial hypertension, or PAH. Although all responded, some with particular gene mutations failed to respond as well,…

Pulmonary hypertension in patients with sarcoidosis is associated with poor prognosis, despite treatment with a targeted therapy, a new study shows. The study “Management and long-term outcomes of sarcoidosis-associated pulmonary hypertension,” was published in the European Respiratory Journal. Patients with sarcoidosis frequently develop pulmonary hypertension. In…

Male gender and lung function parameters have been identified as risk factors for progression of pulmonary arterial hypertension in patients with systemic sclerosis-PAH (SSc-PAH), a study shows. Part of the larger, multi-center, DETECT study, these results are reported in the research, “Factors associated with disease progression in early-diagnosed pulmonary arterial hypertension…

Blocking the enzyme phosphodiesterase 5 (PDE5) is a potential new therapeutic strategy to treat early pulmonary hypertension (PH) in patients who have had a heart attack, a study suggests. The research, “Pulmonary vasodilation by phosphodiesterase 5-inhibition is enhanced and nitric oxide-independent in early pulmonary hypertension after myocardial infarction,”…

The U.S. Food and Drug Administration (FDA) has approved a new, third-generation inhalation device, the TD-300/A, for use with Tyvaso (treprostinil) Inhalation Solution, United Therapeutics Corporation announced. Tyvaso is a prostacyclin vasodilator indicated for the treatment of pulmonary arterial hypertension (PAH) to improve exercise ability. Tyvaso was…

More therapies are now available for the 30 million or so people with rare diseases in the U.S. than ever before, and millions of dollars are being invested in clinical studies that will test new ways of evaluating — and advancing — potential treatments, including the use of natural history…