Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…
High levels of the peptide adiponectin show potential as a biomarker for the prognosis of congenital heart disease in patients with pulmonary hypertension (PH), according to a recent study. Titled “High plasma adiponectin is associated with increased pulmonary blood flow and reduced right…
Endovascular embolization — a minimally invasive surgery that cuts off blood flow from a specific group of blood vessels in the body — can increase the chances of survival in babies with vein of Galen aneurysmal malformation (VGAM) and severe pulmonary hypertension (PH), a study reports. Findings from…
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,…
The EveryLife Foundation for Rare Diseases has launched a scholarship fund in the U.S. to support individuals with rare disorders who are pursuing personal goals through training and education. The initial phase of the five-year, $1-million #RAREis Scholarship Fund will include 32 scholarships — each totaling $5,000 —…
Volibris (ambrisentan) given to treat adults in China with pulmonary arterial hypertension (PAH) caused by connective tissue disease led to significant improvements in exercise capacity without signs of clinical worsening over 24 weeks, a study reported. Improvements were…
A team of researchers have used 3D cell culture technology to develop a model that replicates processes involved in pulmonary arterial hypertension (PAH), allowing them to more carefully analyze the thickening of arterial walls that occurs in patients. Their findings were detailed in the study,…
LIQ861, Liquidia Technologies’ proprietary inhaled dry powder formulation of the vasodilator treprostinil, improves exercise capacity and quality of life for people with pulmonary arterial hypertension (PAH), according to data from the Phase 3 INSPIRE trial. Treatment with LIQ861 also led…
When the COVID-19 pandemic forced the postponement of a rare disease film festival originally slated for May, its organizers set out to find a new way to bring the films to an audience. Co-founders Daniel DeFabio and Bo Bigelow, who are both fathers of children with…
The five-year survival rate is markedly reduced among adults with pulmonary arterial hypertension (PAH) due to congenital heart disease (CHD), compared with other types of CHD, real-world data from the COMPERA-CHD registry show. This worse mortality rate occurred despite the increasing number of PAH-targeted combination therapies now available for…
Aerovate Therapeutics received $72.6 million in Series A funding to advance trials testing AV-101, an experimental inhaled version of imatinib for the treatment of pulmonary arterial hypertension (PAH). AV-101 is an inhaled dry powder that delivers imatinib directly to a patient’s lungs.
To help people experiencing the lingering effects of coronavirus infection, the Pulmonary Wellness Foundation (PWF) has launched a free online rehabilitation and recovery program called COVID Bootcamp 101. In addition to patient education and support, the program offers breathing and other exercises conducted by a team of…
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