The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to a survey conducted by Eurordis-Rare Diseases Europe. “People living with rare diseases in Europe have found themselves caught as collateral…
News
Liquidia Technologies, the developer of an inhaled formulation of treprostinil (LIQ861) for the treatment of pulmonary arterial hypertension (PAH), has completed its acquisition of RareGen, the marketer of generic treprostinil injections. The…
Treatment with Acceleron Pharma’s sotatercept leads to substantial improvements in cardiac parameters, as well as exercise tolerance and capacity, in people with pulmonary arterial hypertension (PAH) on standard therapies, early data from the SPECTRA Phase 2 trial show. The findings, “SPECTRA and Beyond: Signs of Disease Modification?…
Dialing up the activity of a gene called IRF7 may reduce pulmonary artery thickening, lower blood pressure, and ease inflammation in people with pulmonary hypertension (PH), a study using a rat model of the disease suggests. The study, “Downregulation of Interferon regulatory factor 7 is link…
Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about how their respective organizations came to be, as well as the benefits of creating patient registries and how they can help…
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…
Acceleron Pharma announced plans to launch the first Phase 3 clinical trial, called STELLAR, evaluating the safety and effectiveness of its investigational therapy sotatercept (ACE-011) in people with pulmonary arterial hypertension (PAH), before the end of the year. Two other Phase 3 trials testing sotatercept as an…
An increase in right atrial blood pressure during exercise in people with pulmonary hypertension (PH) reflects problems with blood flow in the heart and is linked to a worse prognosis and higher mortality, a study shows. Researchers therefore suggest that right atrial blood…
Off-label use of prostacyclin therapy successfully treated five newborns with persistent pulmonary hypertension of the neonate (PPHN), and switching from intravenous infusion to subcutaneous injection was well tolerated, a case series reported. Its authors recommend larger studies to…
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…
