by CheckRare Staff | Jul 14, 2026
Primary IGF-1 Deficiency Robert Rapaport, MD, Professor of Pediatric Endocrinology, and Director of the Comprehensive Growth Center at the Icahn School of Medicine, Mount Sinai Medical Center, New York City, discussed the differences between severe, primary IGF-1...
by CheckRare Staff | Jul 1, 2026
An Expert Panel on the Evolution of SMA Care Spinal muscular atrophy (SMA) has undergone a remarkable transformation over the past decade. Drs. Nancy Kuntz, Alicia Henriquez, and Angela Lek discuss how advances in disease-modifying therapies have fundamentally changed...
by CheckRare Staff | Jul 1, 2026
Congenital adrenal hyperplasia (CAH) is a group of rare autosomal, recessive genetic disorders that impair production of cortisol (also known as hydrocortisone).[1,2] The hypothalmus and pituitary gland are prompted to overproduce corticotropin-releasing hormone (CRH)...
by CheckRare Staff | Jun 24, 2026
Plasminogen is a precursor protein that is converted into plasmin. Plasmin is crucial to break down fibrin, the main protein component of blood clots.[1] When the body does not produce sufficient plasminogen or does not produce adequate active plasminogen, fibrin can...
by CheckRare Staff | Jun 17, 2026
Lauren Shea, MD, Assistant Professor of Hematology and Oncology, University of Alabama, Birmingham, discusses important aspects of medical treatment and stem-cell transplantation in patients with cutaneous T-cell lymphoma (CTCL). When faced with a...
