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…
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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…
Combined with sildenafil, the potential therapy NTP42 eased symptoms of pulmonary arterial hypertension (PAH) in a rat model of the disease, a study reported. Further testing with other approved treatments is warranted, its researchers wrote, as the paired treatment showed more benefit than either sildenafil (sold as Revatio,…
A bike competition to educational meetings and daily walks, runs, or meditations are among the events Team PHenomenal Hope is hosting throughout November to honor pulmonary hypertension (PH) awareness month. People are invited to join the nonprofit group’s Tour of PHenomenal Hope and its monthlong series, ranging from educational sessions, physical…
A first patient has been dosed in a Phase 1b trial evaluating the safety and efficacy of GMA301, an investigational antibody for the treatment of pulmonary arterial hypertension (PAH), Gmax Biopharm, the therapy’s developer, announced in a press release. GMA301 is a humanized monoclonal antibody,…
The Pulmonary Hypertension Association (PHA) is promoting several events throughout November — the Pulmonary Hypertension (PH) Awareness Month. This year, people are encouraged to discover the risk factors and symptoms of PH, and the challenges faced by those living with the disease, and their families. PH…
