by Madaline Spencer | Jan 22, 2026
Recently, results from a phase 2a study evaluating SGX945 (dusquetide) for the treatment of Behçet’s disease were published in Rheumatology (Oxford). Behçet’s disease is a rare vasculitis characterized by inflammation and swelling of blood vessels. It often affects...
by Madaline Spencer | Jan 21, 2026
Janet Legare, MD, Professor of Pediatrics in the Division of Genetics at the University of Wisconsin, discusses week 52 results of TransCon CNP (navepegritide) in children with achondroplasia. Achondroplasia is a genetic disorder that prevents the...
by Madaline Spencer | Jan 20, 2026
Catherine M. Broome, MD, Professor of Medicine and Director of Cellular Apheresis, Lombardi Cancer Center, MedStar Georgetown University, discusses real world safety data of Enjaymo (sutimlimab) in patients with cold agglutinin disease (CAD). CAD is a rare type...
by Madaline Spencer | Jan 19, 2026
David Kuter, MD, DPhil, Professor of Medicine at Harvard Medical School, Massachusetts General Hospital, discusses treatment with rilzabrutinib in patients with immune thrombocytopenia (ITP). ITP is an autoimmune bleeding disorder characterized by too few...
by Madaline Spencer | Jan 16, 2026
Anita Hill, MD, PhD, Global Medical Head for Hematology & Nephrology and Transplant at Alexion, AstraZeneca Rare Disease, discusses recent data on ravulizumab in rare hematologic conditions. Thrombotic Microangiopathies Following Hematopoietic Stem Cell...
