Patients with pulmonary arterial hypertension (PAH) who carry a specific mutation in the RNF213 gene have poor clinical outcomes and worse response to combination treatments, according to a study from Japan. The study, “Poor outcomes in carriers of the RNF213 variant (p.Arg4810Lys) with pulmonary arterial…
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The incidence of pulmonary arterial hypertension (PAH) is rare in France, but it carries a high economic burden, a new study showed. The data were reported in “Hospital Burden of Pulmonary Arterial Hypertension in France,” a study published in the journal PLOS ONE. Economic studies on the…
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
Analysis of a North American pediatric registry revealed significant variability of pulmonary hypertension (PH) subtypes among children of different racial and ethnic groups, with reduced survival rates among black children with lung disease-associated PH, a new study reports. The study, “Racial…
V-Wave’s new interatrial shunt — a medical device designed to reduce arterial heart pressure — has been granted breakthrough device designation by the U.S. Food and Drug Administration (FDA) for the treatment of people…
Translate Bio is focusing its efforts on the development of new therapies targeting pulmonary diseases, including pulmonary arterial hypertension and cystic fibrosis, among others. In light of this, the company is discontinuing the development of its lead candidate MRT5201 for ornithine transcarbamylase deficiency — a metabolic…
Altavant Sciences presented its patient-centered approach to drug development, which informed the design of an ongoing Phase 2 study, at the Pulmonary Hypertension Association’s recent Annual PH Professional Network (PHPN) Symposium. The effort was detailed in the poster “A Framework for Engaging PAH Patients in…
Owlstone Medical announced that it has introduced a new ReCIVA Breath Sampler and CASPER Portable Air Supply, designed to support researchers working to identify disease biomarkers and to measure levels of known volatile organic compounds (VOCs) on a person’s breath. Confirmed biomarkers provide valuable information to both clinicians and researchers…
The Endoarterial Biopsy Catheter (EABC), a tool for performing biopsies on lung artery walls to better evaluate pulmonary arterial hypertension (PAH) in people and responses to treatment, has been designated a humanitarian use device (HUD) by the U.S. Food and Drug Administration (FDA), its maker, Vascular Biosciences,…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to sotatercept for the treatment of pulmonary arterial hypertension (PAH), according to…
The U.S. Food and Drug Administration (FDA) has agreed to review a request to approve Trevyent (treprostinil) as a treatment of pulmonary arterial hypertension (PAH), United Therapeutics announced in a press release. An FDA decision on this updated New Drug Application (NDA) for Trevyent is expected on or before…
For two-and-a-half painful years, Jerome Maharaj, 42, had no idea what was wrong with him. Born in South Africa, he came to Israel in 1996 and settled in Ma’agan, a small kibbutz near the Sea of Galilee. He got married and raised three children. Jerome Maharaj standing…
