News

Changes in the epigenetic profile — a collection of marks that turn genes on and off  — of pulmonary arterial endothelial cells from people with pulmonary arterial hypertension (PAH) may contribute to disease development, a study suggests. The study, “Remodeling of active endothelial enhancers is associated with aberrant…

While the ongoing COVID-19 pandemic won’t have much of an impact on cash available for new biotech startups, it has begun to cause delays in the development of gene therapies to treat a variety of rare diseases. That’s the consensus of industry experts who spoke in a May 26 webinar…

Kalytera Therapeutics intends to acquire Salzman Group, a company developing R-107 — a liquid form of nitric oxide — to possibly treat pulmonary arterial hypertension (PAH), lung disease associated with COVID-19, and other lung disorders, the company announced. Its acquisition of the Massachusetts-based company should be completed by…

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…