The Pulmonary Hypertension Association (PHA) will be participating in a fundraiser for World PH Day on May 5. World PH Day is an annual event that started in 2012 and seeks to raise awareness about PH. Around the world, more than 80 organizations — including the…
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While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…
FFF Enterprises and Bionews announced today that both rare and orphan disease advocates are joining forces to provide patients with resources to help them connect as a community and continue to manage their health during this time when many are finding themselves alone. Recognized as the nation’s leading supplier of…
United Therapeutics’ Orenitram (treprostinil) improves heart function and lowers risk status in people with pulmonary arterial hypertension (PAH) who had begun treatment with another approved therapy, new data from a Phase 3 clinical trial show. Moreover, compared with Actelion’s Uptravi (selexipag), treatment with Orenitram was linked to significantly…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to CS1, Cereno Scientific’s lead therapy candidate for the treatment of pulmonary arterial hypertension (PAH). CS1 is a unique reformulation of valproic acid, an anti-seizure medication that has been used for…
The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible to…
Newly identified mutations that contribute to a higher susceptibility of idiopathic pulmonary arterial hypertension (IPAH) in people from China, were found in a pathway commonly targeted by therapies that dilate blood vessels (vasodilators), a new study reports. The authors suggested that screening for these mutations…
Based on recently conducted surveys, we know there is a subset of patients with pulmonary hypertension who are currently not on therapies approved by the U.S. Food and Drug Administration to treat their pulmonary hypertension. There is a wide range of possible reasons.
Tricumed‘s LENUS Pro pump, an implantable device approved in Europe for the long-term intravenous administration of Remodulin (treprostinil) to treat pulmonary arterial hypertension (PAH), does not maintain a steady flow rate over time, a study of refill data reports. The analysis found flow rates lower than those specified by…
Small vesicles, called exosomes, released by mesenchymal stem cells helped to ease vascular remodeling in a rat model of pulmonary hypertension (PH), a study reports. The study “Mesenchymal stromal cell-derived exosomes improve pulmonary hypertension through inhibition of pulmonary vascular remodeling” was published in the journal …
The U.S. Food and Drug Administration (FDA) has accepted for review the new drug application (NDA) Liquidia Technologies submitted earlier this year requesting marketing approval of LIQ861, its proprietary inhaled dry powder formulation of treprostinil, for treating pulmonary arterial hypertension (PAH). The U.S.
The U.S. Food and Drug Administration (FDA) has granted the designation of breakthrough therapy to Acceleron Pharma‘s investigational molecule sotatercept for the treatment of pulmonary arterial hypertension (PAH). Breakthrough designation is given to medications that have the potential to provide…
