The Rare Disease Report page is a resource for medical news and expert insights on rare diseases. This page features expert-led coverage, articles, videos and research on the therapies and development of treatments for sickle cell disease, multiple myeloma, and more.
April 29th 2024
FDA approves mavorixafor (XOLREMDI) for WHIM syndrome, the first approved treatment for the rare disease.
From Early Hypothesis to Clinical Data About Reducing Vaso-Occlusive Episodes with Arginine Therapy
December 22nd 2022Almost 25 years later, Dr. Claudia Morris concluded 2022 by presenting phase 2 data at the 64th ASH Annual Meeting that demonstrated the therapy's benefit, and her original premise. Her team's phase 3 trial evaluating Arg is currently underway.
Corinna Schultz, MD: Does Sickle Cell Trait get Addressed?
December 12th 2022"There's no national guidance on how this information should be delivered back to families, which leaves it up to each state's newborn screening program to decide the best route that they see fit," Dr. Corinna Schultz explains.