The State of Rare Diseases

  Richard A. Basile, Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses the state of rare diseases and the critical challenges facing it. BioPontis is an international nonprofit organization whose mission is to advance promising science into...

FDA Approval of Revcovi for Treating ADA-SCID Patients

The FDA approved Revcovi (elapegademase-lvlr) injection in the United States, for the treatment of adenosine deaminase severe combined immune deficiency (ADA-SCID) in pediatric and adult patients. “We are gratified by the FDA’s timely recognition of...

Cystic Fibrosis and Pulmonary Exacerbations

  Greg Duncan, CEO of Celtaxsys discusses cystic fibrosis (CF) and the role of pulmonary exacerbations. Celtaxsys recently announced top line results of its Phase 2 EMPIRE-CF trial evaluating oral, once daily anti-inflammatory molecule, acebilustat, for the...

FDA Approves Antibacterial Drug for Rare Lung Disease

The U.S. Food and Drug Administration approved a new drug, Arikayce (amikacin liposome inhalation suspension), for the treatment of lung disease caused by a group of bacteria, Mycobacterium avium complex (MAC) in a limited population of patients with the disease who...
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