Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…
News
The first person with coronavirus disease 2019 (COVID-19) complicating pulmonary hypertension (PH) has received at-home treatment with inhaled nitric oxide (iNO), delivered through the Genosyl system, according to VERO Biotech, manufacturer of Genosyl. The PH patient with COVID-19 infection was treated under the terms of an emergency…
People with connective tissue disease-associated pulmonary arterial hypertension (CTD-PAH) — particularly those showing low or intermediate disease risk, typical PAH — benefit most from combination therapy with Letairis (ambrisentan) and Adcirca (tadalafil) than from either therapy alone, according to an exploratory sub-analysis of a Phase 3…
Treatment with Orenitram (oral treprostinil) is associated with lower healthcare-related costs than treatment with Uptravi (selexipag) in people with pulmonary arterial hypertension (PAH), a new study suggests. The study, “Medication Adherence and Healthcare Costs Among Patients with Pulmonary Arterial Hypertension Treated with Oral Prostacyclins: A Retrospective…
Among people with chronic thromboembolic pulmonary hypertension (CTEPH), certain anticoagulants may reduce the risk of serious bleeding more than others, a new study shows. The study, “Real-life data of direct anticoagulant use, bleeding risk and venous thromboembolism recurrence in chronic thromboembolic pulmonary hypertension patients: an observational…
The Phase 2 HELP study testing levosimendan as a potential treatment for pulmonary hypertension (PH) has exceeded its target number and is fully enrolled, Tenax Therapeutics announced. The trial (NCT03541603), testing the potential therapy in PH patients with heart failure…
Bellerophon Therapeutics has reached an agreement with the U.S. Food and Drug Administration (FDA) setting the design of an upcoming, pivotal Phase 3 clinical trial testing INOpulse, an investigational therapy, in people with pulmonary fibrosis (PF) who are at risk of pulmonary hypertension (PH). PH-PF is characterized…
The number of both new and existing cases of pulmonary arterial hypertension (PAH) in the southwestern U.S. population was found in a recent study to be higher than that reported in the largest U.S. PH registry, called REVEAL, or the Registry to Evaluate Early and Long-term PAH.
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…
Genetic screening of patients with persistent pulmonary hypertension of the newborn (PPHN) found three rare mutations associated with the condition, suggesting it has a genetic basis, a study reports. The researchers also found that a link between PPHN and genetic variants…
Men with pulmonary arterial hypertension (PAH) are more likely to carry mutations in the BMPR2 gene, a known genetic risk factor for PAH, a study from China found. Male PAH patients had more severe disease and were diagnosed at younger age than women. The results support the therapeutic potential…
In recognition of Rare Disease Day Feb. 29, Bionews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the #WhatMakesMeRare campaign was aimed at uplifting people with rare diseases by encouraging them to share their stories and perspectives. The…
