News

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

The National Institutes of Health (NIH) awarded a $2.8 million Fast Track Small Business Innovation Research (SBIR) grant to PhaseBio Pharmaceuticals to advance the clinical development of PB1046, the first sustained-release analogue of the natural vasoactive intestinal peptide (VIP), in patients with pulmonary arterial hypertension (PAH). In…

Many people with chronic kidney disease also have pulmonary hypertension, a condition that increases their risk of heart problems and death, a review of 16 studies found. Doctors refer to health problems that can damage the heart muscle as cardiovascular events. They include a heart attack, an irregular heartbeat, or a…

A new cardiac surgical procedure to treat mitral valve disease, which is minimally invasive, does not require open-heart surgery, and offers faster recovery, is being tested in an upcoming clinical trial.  Mitral valve disease occurs when the heart’s mitral valve — which has…

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

Subcutaneous Remodulin (treprostinil), marketed by United Therapeutics, is to be safe and effective for at least 12 months in patients with congenital heart disease-related pulmonary arterial hypertension (CTD-PAH), an observational study found. The study, “Subcutaneous treprostinil in congenital heart disease-related pulmonary arterial hypertension,” was published in the…

Development of pulmonary hypertension (PH) in cystic fibrosis (CF) patients compromises lung function and blood-gas parameters, but does not significantly worsen survival, an analysis suggests. The study, “Prognostic significance of pulmonary hypertension in patients with cystic fibrosis: A systematic review and meta-analysis,” was published in the journal …

A rock-painting contest in Las Vegas. A fashion show in New York. A 7,000-meter race around the Washington Monument that’ll coincide with a similar #Racefor7 event in Bengaluru and Mumbai, India. From Athens to Atlanta, from San Diego to Sydney, people across the globe will mark World Rare Disease…

The presence of systemic autoimmune rheumatic diseases (ARDs) — a comorbidity that can range from systemic sclerosis to Sjögren’s syndrome — does not impact survival rates among adults with pulmonary arterial hypertension (PAH) who undergo a lung transplant, a study says. The report, “Survival of…

Treatment with Tracleer (bosentan) is effective in patients with pulmonary arterial hypertension (PAH), a new review study and analysis of data from clinical trials shows. The research, “Bosentan Therapy for Pulmonary Arterial Hypertension and Chronic Thromboembolic Pulmonary Hypertension: A Systemic Review and…

Opsumit (macitentan) failed to improve the cardiac function of people with pulmonary hypertension stemming from a heart problem, a Phase 2 clinical trial shows. The PH patients who took it were also at increased risk of fluid retention, researchers said. The therapy’s developer, Actelion, reported the results in the European…

Reata Pharmaceuticals announced that pulmonary arterial hypertension (PAH) patients treated with bardoxolone methyl showed significant improvements in kidney function that were sustained for two years without adverse outcomes. The results from the long-term follow-up of the Phase 2 LARIAT study (NCT02036970) showing a two-year improvement in kidney…