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…
Balloon pulmonary angioplasty (BPA) is a safe and effective, minimally invasive treatment for people with inoperable chronic thromboembolic pulmonary hypertension (CTEPH), according to a study from the U.K. The study describes the treatment of the first group of CTEPH patients to be treated with BPA.
A consensus on the use of oral prostacyclin pathway agents (PPAs), like Uptravi (selexipag), as an add-on to standard therapies for certain types of pulmonary arterial hypertension (PAH) was announced by a 19-physician expert panel. PPAs can be an adjunct therapy to standard vasodilator compounds — endothelin…
Reata Pharmaceuticals is stopping clinical trials evaluating bardoxolone methyl in people with pulmonary arterial hypertension (PAH), including a Phase 3 study, to ensure the safety of participants during the COVID-19 pandemic, the company announced. Trials in other disorders are set to continue, although some will stop…
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD), said it will still take place at the…
A 62-year-old with systemic sclerosis-associated pulmonary arterial hypertension (SSc-PAH) was successfully treated with immunosuppressants and vasodilators, a case study reports. The unusual antibody linked to her disease, called the anticentriole antibody, may also serve as a marker for the use of immunosuppressives in SSc-PAH patients with this antibody. The…
At 3 a.m. on July 6, 2003, Miami resident Jeffrey Goldstein got a call that would change his life. Doctors at the Duke University Lung Transplant Clinic had found a lung donor match for Goldstein, who had been diagnosed with pulmonary fibrosis two years earlier. Nearly 17 years…
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