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...
by Madaline Spencer | Jan 15, 2026
A recent paper published in Molecular Genetics and Metabolism presented safety and efficacy outcomes from the 48-month open-label extension of the NPC-002 study of arimoclomol in patients with Niemann-Pick type C (NPC). NPC is a complex lipid storage disease caused by...
by Madaline Spencer | Jan 14, 2026
Jan Klatt, MD, Head of Development Unit Neurology & Immunology, Merck Healthcare, Germany, discusses the U.S. Food and Drug Administration’s (FDA) Fast Track designation of cladribine capsules for patients with generalized myasthenia gravis (gMG). ...
by Madaline Spencer | Jan 13, 2026
The U.S Food and Drug Administration (FDA) has approved Zycubo (copper histidinate) for the treatment of pediatric patients with Menkes disease. Menkes disease is a rare X-linked pediatric disease caused by gene mutations of copper transporter ATP7A. It affects...
