It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
News
Into the vein (intravenous) injections of Revatio (sildenafil) have no added benefit over inhaled nitric oxide (iNO) used alone in treating newborns with persistent pulmonary hypertension (PPHN), a Phase 3 trial sponsored by Pfizer shows. Revatio, marketed by Pfizer, was ineffective as an intravenous formulation combined…
Use of sequential combination therapy focused on changes in exercise capacity improves health-related quality of life (HRQoL) in patients with newly diagnosed pulmonary arterial hypertension (PAH), according to a new study. The research, “Medium-term health-related quality of life in patients with pulmonary arterial hypertension treated with…
Treatment with udenafil (brand name Zydena) can increase exercise capacity and is well-tolerated in people with pulmonary arterial hypertension (PAH), results from a Phase 2b clinical trial show. The trial data was reported in the study “Efficacy and Safety of Udenafil for the Treatment of Pulmonary Arterial…
Acceleron Pharma has reached its patient enrollment goal in the ongoing Phase 2 PULSAR trial, with more than 100 participants. The trial is testing the safety and effectiveness of sotatercept (ACE-011), the company’s investigational therapy for the treatment of people with pulmonary arterial hyptertension (PAH). The…
Philips recently announced two new capabilities of its echocardiogram system, the EPIQ CVx cardiology ultrasound platform, that will assist in heart imaging and functional studies, improving the accuracy and duplication of diagnostic tests for pulmonary hypertension (PH), among other conditions. The system improvements are the result…
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…
Adverse effects are associated with poor adherence to phosphodiesterase-5 inhibitors (PDE-5I) as treatment for patients with pulmonary arterial hypertension (PAH) treated via an integrated care model at an academic institution. Hospitalization events, out-of-pocket costs and frequency of dosing did not play a role, a study shows. Led by researchers at…
A new generic version of Actelion’s Tracleer (bosentan) developed by Teva Pharmaceutical was approved by the U.S. Food and Drug Administration (FDA) for the treatment of pulmonary arterial hypertension (PAH). The decision was supported by evidence that the generic bosentan has similar biochemical properties to…
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, find assistance and receive treatment. Eurordis-Rare Diseases Europe hopes to improve the current piecemeal treatment and support program with a holistic,…
A new meta-analysis assessing balloon pulmonary angioplasty (BPA) and Adempas (riociguat) for treating inoperable chronic thromboembolic pulmonary hypertension (CTEPH) found both treatment strategies to be well-tolerated, and able to improve exercise tolerance and the function of the circulatory system. The study with that finding, “Balloon…
Note: This is the second article in a three-part series written in collaboration with respiratory therapist Mark W. Mangus Sr., RRT, RPFT, FAARC, and oxygen expert Ryan Diesem. The first part can be found here. When it comes to home improvement, people typically want three things from a contractor:…
