Marisa Wexler, MS,  senior science writer—

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…

The European Commission has granted orphan drug designation to sotatercept for the treatment of people with pulmonary arterial hypertension (PAH). This designation is given to medications with the potential to substantially improve care for rare diseases — defined as those that affect fewer than five individuals per 10,000…

Enrollment is resuming for the VIP clinical trial, which is evaluating PhaseBio Pharmaceuticals‘ investigational therapy pemziviptadil (PB1046) in people with pulmonary arterial hypertension (PAH). The trial, which is recruiting adult patients at sites across the U.S., underwent a brief pause in enrollment owing to effects of the…

Beyond Air filed an application with the U.S. Food and Drug Administration (FDA) requesting approval of its LungFit PH system — an investigational device used to deliver the vasodilator nitric oxide — to treat persistent pulmonary hypertension of the newborn (PPHN). FDA guidelines allow a 180-day (about…

Mayo Clinic and Vocalis Health announced a collaboration to research and develop new voice-based tools for screening, detecting, and monitoring health. The collaboration will begin with a project that seeks to identify vocal biomarkers for pulmonary hypertension (PH); such markers could help healthcare providers detect and treat the…

The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases.  “The RareLaunch program is…

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…

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…

The combination of Opsumit (macitentan) and Adcirca (tadalafil) is a safe and effective initial treatment for pulmonary arterial hypertension (PAH), new clinical trial data show. The findings were published in the European Respiratory Journal, in the study, “Initial combination therapy of macitentan and tadalafil…

The U.S. Food and Drug Administration (FDA) has accepted for review the  application for Tyvaso (inhaled treprostinil) as a potential treatment for pulmonary hypertension associated with interstitial lung disease (PH-ILD). The supplemental new drug application (sNDA) was submitted by United Therapeutics,…