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...
by Madaline Spencer | Jan 13, 2026
Floyd Stewart, patient with stage 4 nasopharyngeal carcinoma, and Monique Stewart, advocate and Floyd’s wife, discuss their family’s experience with the disease and their advocacy work. Nasopharyngeal carcinoma is a rare head and neck cancer arising from...
