News

A photo of a bespectacled young boy, his red baseball cap slightly askew as he  enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…

Treatment with Tyvaso (inhaled treprostinil) can improve lung function in people with pulmonary hypertension associated with interstitial lung disease (PH-ILD), new data from a completed Phase 2/3 clinical trial show. These data were presented at CHEST 2020, the annual meeting of the American College of…

A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its use in future appointments, a series of surveys found. The surveys were conducted by the National Organization for Rare Disorders (NORD) and involved more than 800…

The combination of pulmonary endarterectomy (PEA), a surgery to remove blood clots in the lungs, and balloon pulmonary angioplasty (BPA), the use of balloons to open narrowed or blocked blood vessels — could improve outcomes for people with chronic thromboembolic pulmonary hypertension (CTEPH), researchers reported. Treatment, including…

Tenax Therapeutics  has acquired developmental and commercial rights for North America to an oral formulation of levosimendan, a potential therapy for pulmonary hypertension and heart failure with preserved ejection fraction (PH-HFpEF). Tenax plans to open a Phase 3 safety and efficacy trial of…

Suppressing CD248, a cell surface protein, reduced pulmonary vascular remodeling — the key structural alteration in pulmonary arterial hypertension (PAH) — in a rat model of the disease. CD248 was found to promote PAH-associated abnormal growth and migration of pulmonary artery smooth muscle cells (PASMCs), the cells that line the…

The U.S. Food and Drug Administration (FDA) has granted SoniVie permission to begin a pivotal clinical trial of its Therapeutic Intra-Vascular Ultrasound (TIVUS) System in people with pulmonary arterial hypertension (PAH). This “study will provide important information on the safety and efficacy of the TIVUS System in…

New variants in the DNA region that promote the activity of the BMPR2 gene — associated with familial pulmonary arterial hypertension (PAH) — lower the gene’s activity and may contribute to the condition in some families, a study…

Elevated levels of the protein IGFBP2 were identified in the blood of people with pulmonary arterial hypertension (PAH) and found to be associated with lower physical function, higher arterial blood pressure, and an increased mortality risk, a study reports.  …

Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…

For the first time, the long-term use of antipsychotic medications has been linked to the development of chronic thromboembolic pulmonary hypertension (CTEPH), a case study reports.  The authors recommend that CTEPH should be considered in patients who receive antipsychotics and also have symptoms associated…

An imbalance in the composition of bacteria living in the gut, a phenomenon known as dysbiosis, may play a key role in the onset and progression of pulmonary arterial hypertension (PAH), a study in animals suggests. The study, “Gut microbiota modification suppresses the development of…