News

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons and…

Statins Found to Benefit Patients with 2 Chronic Lung Disorders

Statins have protective effects and can benefit patients with both pulmonary hypertension (PH) and chronic obstructive pulmonary disease (COPD), according to a large retrospective study in Taiwan. The study, “Statins for the Treatment of Pulmonary Hypertension in Patients with Chronic Obstructive Pulmonary Disease,” was published…

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Pemziviptadil Shows Promise in Small Clinical Trial

PhaseBio Pharmaceuticals‘s investigational therapy pemziviptadil (PB1046) showed a good safety profile and clinically meaningful effects on physical function in a small clinical trial of three people with pulmonary arterial hypertension (PAH). The findings support further evaluation of pemziviptadil as…

ERN-LUNG Project Helping People With Rare Lung Diseases

“Patient journeys” is an initiative designed to help patients and clinicians  improve care for rare lung diseases, including pulmonary hypertension (PH). The project, announced in a press release from the European Reference Network on Rare Respiratory Diseases (ERN-LUNG), presents testimonies showing the clinical history and needs of patients through…

Protein Could Help Monitor Heart Stress in Rare Form of PH

Soluble ST2 (sST2) protein could be an additional non-invasive biomarker for monitoring patients with chronic thromboembolic pulmonary hypertension (CTEPH) undergoing interventional treatment with balloon pulmonary angioplasty (BPA), a study suggests. The study, “Soluble ST2 as a Biomarker for Early Complications in Patients with Chronic Thromboembolic Pulmonary Hypertension Treated…