Marisa Wexler, MS,  senior science writer—

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,…

High pulmonary vascular resistance (PVR) is associated with worse mortality rates among people with pulmonary hypertension (PH), and the bottom threshold for what constitutes “elevated” PVR is lower than previously thought, a new study suggests. “The findings of this study cast the clinical profile of patients who have PH and…

People with pulmonary arterial hypertension (PAH) who are treated at specialty care centers have better survival chances and a lower likelihood of hospitalization, a study reports. These findings support referring PAH patients to specialty care centers for treatment, where factors like more careful disease monitoring might be decisive. The…

U.S. veterans with pulmonary hypertension (PH) due to left heart disease and chronic lung disease are more likely to be inappropriately prescribed phosphodiesterase-5 inhibitors (PDE5i) if they have received care both in and out of the Veterans Health Administration (VA), a study has found. Other factors for…

At-home treatment with inhaled nitric oxide through Genosyl, a tankless delivery system, was used successfully to manage pulmonary arterial hypertension (PAH) complicated by COVID-19 in a patient, according to a case study. The case was described in the report “Outpatient Inhaled Nitric Oxide in a…

LIQ861, Liquidia Technology‘s investigational inhaled formulation of treprostinil, is safe and well-tolerated in patients with pulmonary arterial hypertension (PAH), according to final results from a Phase 3 clinical trial. The trial results supported the new drug application…