European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a May 14-15 online medical conference. The session was part of the 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was to have occurred…
Chronic inhalation of nicotine, the addictive component of all tobacco products, is enough to trigger changes in pulmonary arteries and in the heart’s right chamber that can cause pulmonary hypertension (PH), a recent study in mice found. The study, “Effects of Chronic Nicotine Inhalation on…
Newly published research has detailed the underlying biochemical process behind Acceleron Pharma’s investigational therapy sotatercept (ACE-011) for the potential treatment of people with pulmonary arterial hypertension (PAH). The findings, in two rat models of PAH,…
Eurordis, a Paris-based coalition of national rare disease associations across Europe, hosted its first all-virtual conference, bringing some 1,500 delegates from 57 countries together online during the COVID-19 pandemic. The 10th European Conference on Rare Diseases & Orphan Products (ECRD2020) — which was set for May 14–15 in…
Obesity is linked to a lower risk of mortality in patients with pulmonary hypertension (PH), particularly among those with precapillary PH, a study found. The study, “Obesity Is Associated With Pulmonary Hypertension and Modifies Outcomes,” was published in the Journal of the American…
Mallinckrodt Pharmaceuticals’ INOmax (nitric oxide) gas improves oxygenation in premature newborns with pulmonary hypertension (PH) at least as effectively as it does in term and near-term babies, data from a Phase 4 observational study show. These findings, from a second planned interim study analysis (at 75% enrollment),…
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…
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…
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