News

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

Patients with elevated pulmonary arterial pressure, but not high enough to be diagnosed with pulmonary hypertension (PH), have a poorer prognosis compared to those with lower pulmonary arterial pressure. According to the findings of the study, “Prognostic Effect and Longitudinal Hemodynamic Assessment of Borderline Pulmonary Hypertension,”…

Preclinical work supports the potential of  sotatercept to be a first-in-class disease-modifying therapy for pulmonary arterial hypertension (PAH) and to moving the investigational therapy into a Phase 2 trial in patients, Acceleron Pharma announced. Sotatercept is a  trap molecule for members of the transforming growth factor-beta (TGF-beta) superfamily. A growing body of evidence…

Vivus announced that it is in discussions with the U.S. Food and Drug Administration (FDA) to possibly test tacrolimus — an oral medication used to prevent organ rejection after a transplant — as a potential treatment for pulmonary arterial hypertension (PAH). In a scheduled pre-Investigational New Drug (IND) application meeting, the…

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…