by CheckRare Staff | Aug 1, 2023
Scott Schobel, MD, Chief Medical Officer at Vico Therapeutics, discusses current treatment options for Huntington’s disease and spinocerebellar ataxia (SCA1 and SCA3). Huntington’s disease is a rare neurodegenerative disorder characterized by...
by CheckRare Staff | Jul 31, 2023
Binod Dhakal, MD, Associate Professor at the Medical College of Wisconsin discusses current treatment options for patients with refractory or relapsed multiple myeloma. Multiple myeloma is a rare blood cancer associated with uncontrolled growth of plasma...
by CheckRare Staff | Jul 30, 2023
WHIM syndrome is a rare, congenital primary immunodeficiency disorder associated with neutropenia that typically presents in childhood or adolescence, predominantly caused by pathogenic variants in the CXCR4 chemokine receptor gene.[1] The CXCR4 receptor is involved...
by CheckRare Staff | Jul 28, 2023
Jennifer Puck, Professor of Pediatrics at the University of California, San Francisco, and co-leader of Rare Diseases Clinical Research Network’s (RDCRN) Primary Immune Deficiency Treatment Consortium, and Monica Thakar, MD, Associate Professor at both Fred...
by CheckRare Staff | Jul 19, 2023
Richard Lafayette, MD, FACP, Director of the Stanford Glomerular Disease Center, and Rheumatologist at Stanford Health Care, describes the latest clinical trial assessing Nefecon (budesonide delayed-release capsules) for immunoglobulin A (IgA) nephropathy treatment....
