The Oncologic Drugs Advisory Committee (ODAC) of the U.S. Food and Drug Administration (FDA) voted unanimously (11 – 0) in favor of approving tazemetostat to treat patients with epithelioid sarcoma.
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.
Yuliya Linhares, MD, Chief of Lymphoma Services at Miami Cancer Institute, discusses strategies for shortening the diagnostic journey of CTCL.
Rett syndrome is a multisystem disorder that primarily affects girls; multiple loss-of-function mutations to the MECP2 gene are the cause of this rare disease.
Aleena Banerji, MD, highlights the current and future treatment options for patients with hereditary angioedema (HAE).
