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.
Phase 4 Trial Shows Positive Results for Biweekly Dosing of Xembify in Primary Immunodeficiencies
July 24th 2023Investigators aimed to support extending the US FDA labeling of Xembify to include the biweekly dosing option, therefore, providing greater flexibility and convenience for patients managing their immunodeficiencies.
Guy Young, MD: Prophylactic or Gene Therapy, and Shared Decision Making in Hemophilia
July 8th 2023Informed discussions about the pros and cons of different treatments help guide the decision-making process. When it comes to treatment adherence and overall quality of life, patient lifestyle plays a significant role.