Hemophilia A Treatment Options | CheckRare.com
Snejana Krassova, MD of Bayer Pharmaceuticals talks about the need for more treatment options to be available for persons with hemophilia A.
Snejana Krassova, MD of Bayer Pharmaceuticals talks about the need for more treatment options to be available for persons with hemophilia A.
Larisa Geskin, MD, Columbia University Medical Center and Comprehensive Skin Cancer Center at the Division of Cutaneous Oncology in the Department of Dermatology, discusses the…
Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare, genetic disorder that affects the nervous and musculoskeletal systems of young patients.
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…
Nicholas Silvestri, MD, of the Univ of Buffalo, provides an overview of the latest myasthenia gravis clinical research presented at AAN 2024.
Learn new treatment strategies for MPSs, Niemann-Pick type C and lesser know LDs.
Cutaneous T-cell lymphoma (CTCL) belongs to the non-Hodgkin lymphoma class of hematologic T-cell lymphoproliferative disorders.
WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with severe neutropenia that affects all ages.
Three case studies exploring complexities of diagnosing, treating, and managing patients with various complement-mediated kidney disorders.
Learn about adding a disease to newborn screening panels, and how to interpret NBS results.
Learn the role of protein activators in lysosomal disorders and the role of inflammatory pathways in sphingolipid disorders.