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…
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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…
Tenax Therapeutics has completed its Phase 2 HELP clinical trial testing levosimendan as a potential treatment for patients with pulmonary hypertension and heart failure with preserved ejection fraction (PH-HFpEF). Top-line trial results…
Despite previous evidence to the contrary, a single-center study suggests that sex hormones do not influence the development or progression of pulmonary arterial hypertension (PAH), nor do they explain the higher prevalence of PAH in women. Exposure to sex hormones, either due to external sources like oral contraceptives…
First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…
I’ve written before about how I have adjusted my diet since my pulmonary hypertension diagnosis, but this isn’t something I’ve always found easy. When struggling with mental health or the usual pressures of daily life, it can be…
