Rare Disease Focus: Lung Diseases +++ +++ +

CLINICAL PERSPECTIVES Committee for Medicinal Products for Human Use Recommendation for Sotatercept for the Treatment of Pulmonary Arterial Hypertension https://checkrare.com/committee-for-medicinal-products-for-human-use-recommendation-for-sotatercept-for-the-treatment-of-pulmonary-arterial-hypertension/ Marius Hoeper, MD, Respiratory Physician at Hannover Medical School, Germany, discusses a recent regulatory update by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in the treatment of pulmonary arterial hypertension (PAH). Approval of Nerandomilast Tablets To Treat Patients With Progressive Pulmonary Fibrosis https://checkrare.com/approval-of-nerandomilast-tablets-to-treat-patients-with-progressive-pulmonary-fibrosis/ Shervin Assassi, MD, Director, Division of Rheumatology at McGovern Medical School, discusses the approval of Jascayd (nerandomilast) tablets for treatment of patients with progressive pulmonary fibrosis (PPF). New Formulation of Berotralstat for Treatment of Pediatric Patients With Hereditary Angioedema https://checkrare.com/new-formulation-of-berotralstat-for-treatment-of-pediatric-patients-with-hereditary-angioedema/ Raffi Tachdjian, MD, Associate Clinical Professor of Medicine & Pediatrics, Division of Allergy & Clinical Immunology, University of California Los Angeles, discusses a new formulation of Orladeyo (berotralstat) for the treatment of pediatric patients with hereditary angioedema (HAE). Rare Diseases in Ireland – New Efforts to Improve Access to Care https://checkrare.com/rare-diseases-in-ireland-new-efforts-to-improve-access-to-care/ Each country takes a different approach to rare diseases, from the way it defines the term to the health policies it implements to its approach to research. Data From the CORAL Trial of Nalbuphine ER in Patients With Idiopathic Pulmonary Fibrosis https://checkrare.com/data-from-the-coral-trial-of-nalbuphine-er-in-patients-with-idiopathic-pulmonary-fibrosis/ Data from the Phase 2b CORAL trial of nalbuphine extended release (ER) for the treatment of chronic cough in patients with idiopathic pulmonary fibrosis (IPF) was published in the Journal of the American Medical Association (JAMA). Open-Label Extension Study Testing Arimoclomol in Patients With NPC https://checkrare.com/open-label-extension-study-testing-arimoclomol-in-patients-with-npc/ A recent paper published in Molecular Genetics and Metabolism presented safety and efficacy outcomes from the 48-month open-label extension of the NPC-002 study of arimoclomol in patients with Niemann Pick type C. A Family’s Experience With Nasopharyngeal Carcinoma https://checkrare.com/a-familys-experience-with-nasopharyngeal-carcinoma/ Floyd Stewart, patient with stage 4 nasopharyngeal carcinoma, and Monique Stewart, advocate and Floyd’s wife, discuss their family’s experience with the disease and their advocacy work.