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.
January 27th 2023
The study population consists of patients who received 1-3 prior lines of therapy for relapsed and lenalidomide-refractory multiple myeloma.
January 13th 2023
January 5th 2023
Promising results from the IMerge phase 3 clinical trial met the primary and secondary endpoints in the investigation of imetelstat, a novel telomerase inhibitor developed for hematologic malignancies.
The targeted patient population included those with sickle cell disease or beta thalassemia who were considered at high risk for complications, or otherwise ineligible for standard myeloablative hematopoietic stem cell transplant.
Expert Stephanie Guarino, MD, MSHP, shares her plan for the future of pain management specifically for sickle cell disease is based on results from a series of studies.
"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.
Updated findings from the ongoing clinical trial investigating lovo-cel in sickle cell disease and persistent anemia allude to the possibility that a contributing factor of anemia was identified.