The U.S. Food and Drug Administration (FDA) has approved the first vaccine to prevent Ebola virus disease. The vaccine, Ervedo, by Merck & Co, Inc, was granted approval based on a study conducted during the 2014-2016 outbreak in Guinea. The study involved 3,537 adults who were in contact with people infected by Ebola, or in …
Learn about the challenges of finding suitable candidates for gene therapy clinical trials, and limitations of ERT for MPS.
Marina Kremyanskaya, MD, PhD, discusses the development of gene silencer divesiran for treatment of polycythemia vera (PV).
John Kuruvilla, MD, explores best practices for discussing possible clinical trial participation with patients with hematologic malignancies
Learn about the role of AI in suspecting or diagnosing LDs.
Almost half of all novel medications approved by the U.S. Food and Drug Administration (FDA) are orphan drugs. Below is the list of important regulatory…
Juozas Gordevicius, PhD, Chief Technology Officer of VUGENE Vilnius, Lithuania, discusses autophagy-lysosomal systems in Parkinson’s disease.
David Warnock, MD, Professor of Medicine (Emeritus), University of Alabama at Birmingham, discusses recent advances in Fabry disease.
Erdheim-Chester disease is a rare condition affecting many parts of the body, it most commonly affects adults.
The role of neonatal fragment crystallizable receptor (FcRn) in myasthenia gravis (MG) and treatment options
Joseph E. Sullivan, MD, University of California San Francisco, discusses zovrevunersen for the treatment of Dravet syndrome.
