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.
Vutrisiran Treatment in Patients with Amyloidosis with Polyneuropathy Promising
January 21st 2022The therapeutic met all secondary endpoints measured at 18 months in patients with hATTR amyloidosis with polyneuropathy, which included statistically significant improvements in neuropathy, and quality of life.
Tim Duong, PhD, Wouter Hoogenboom, PhD: What is Known About COVID-19 and SCD
January 17th 2022After conducting and extensive literary review on all eligible data regarding COVID-19 in patients with sickle cell disease or trait, experts discuss what is known and what needs to be better understood about the pandemic's effect on this population.