With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…
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TriAxia Health is partnering with three academic medical centers to build a large database aiming for a better understanding of rare pulmonary diseases, beginning with pulmonary arterial hypertension (PAH). To be meaningful, medical research often requires very large quantities of data. Bigger datasets give a study greater statistical…
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.
Children who undergo bone marrow transplants could develop pulmonary hypertension (PH), which could lead to severe complications if not diagnosed and treated promptly, a retrospective study found. The study, “Pulmonary hypertension after bone marrow transplantation in children,” was published in the European Respiratory Journal.
Cardiopulmonary exercise testing can improve the ability of an algorithm to identify systemic sclerosis patients who have pulmonary arterial hypertension (PAH), a new study suggests. The study, “Cardiopulmonary exercise testing in a combined screening approach to individuate pulmonary arterial hypertension in systemic sclerosis,” was published in…
New preclinical data supports the positive effects of Liminal Biosciences’s lead candidate, PBI-4050, in reducing pulmonary hypertension and the abnormal enlargement of the heart’s right ventricle muscle. The company presented its latest data on two posters at the American Heart Association 2019 conference, held recently in Philadelphia. The…
Actelion Pharmaceuticals has withdrawn all ongoing worldwide requests for expanded approval of Opsumit (macitentan), a pulmonary arterial hypertension (PAH) medicine, to treat adults with inoperable chronic thromboembolic pulmonary hypertension (CTEPH). The decision was not driven by any safety concerns, Actelion, one of …
Like many rare disease charities, the Pulmonary Hypertension Association (PHA) was formed in 1991 by four women sitting around a kitchen table in Florida, hoping for a cure. Yet the PHA is probably the only patient advocacy group that actually preserved its original kitchen table and chairs — and made…
An antibody that recognizes and blocks the activity of osteoprotegerin, a protein involved in the development of pulmonary arterial hypertension (PAH), was able to stop and even reverse disease progression in animal and cell models of PAH, a study reports. The study, “A therapeutic antibody…
Use of Bellerophon Therapeutics’s INOpulse device led to clinically meaningful improvements in people with pulmonary hypertension associated with interstitial lung disease (PH-ILD), according to initial data from an ongoing clinical study. PH-ILD is associated with constriction or narrowing of pulmonary arteries, which supply the lungs with…
Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…
The vasodilator INOpulse increased physical activity and helped maintain higher levels of exercise in people at risk of pulmonary hypertension (PH) associated with pulmonary fibrosis (PH-PF), preliminary results from a Phase 2 trial show. The results were presented in two posters at the Pulmonary Fibrosis Foundation Summit 2019,…
