The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other…
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At-home treatment with inhaled nitric oxide through Genosyl, a tankless delivery system, was used successfully to manage pulmonary arterial hypertension (PAH) complicated by COVID-19 in a patient, according to a case study. The case was described in the report “Outpatient Inhaled Nitric Oxide in a…
The European Medicines Agency (EMA) has granted priority medicines (PRIME) designation to investigational therapy sotatercept (ACE-011) for the treatment of patients with pulmonary arterial hypertension (PAH). This designation is given to medications in development that have the potential to treat conditions with an unmet medical need…
When given alone or in combination with other therapies, Opsumit (macitentan) leads to clinically meaningful improvements in right heart function in people with pulmonary arterial hypertension (PAH), according to data from a final analysis of a Phase 4 trial. Findings were announced in the presentation “…
United Therapeutics has temporarily stopped enrolling patients in ongoing clinical trials of treprostinil-based therapies for pulmonary arterial hypertension (PAH) amid the COVID-19 pandemic, the company announced. Treprostinil is a man-made version of the hormone prostacyclin, a natural compound that helps blood vessels relax and widen, prevents arteries from blocking, and…
LIQ861, Liquidia Technology‘s investigational inhaled formulation of treprostinil, is safe and well-tolerated in patients with pulmonary arterial hypertension (PAH), according to final results from a Phase 3 clinical trial. The trial results supported the new drug application…
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…
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…
