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.
October 10th 2024
Based on the regulatory feedback provided by the FDA, Nanoscope announced plans to initiate Biologics License Application submission in Q1 2025.
September 29th 2024
Expert Illustrations & Commentaries™: Exploring the Role of Novel Agents for the Management of IgA Nephropathy
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‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease
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Clinical ShowCase™: Forming a Personalized Treatment Plan for a Patient With ANCA-Associated Vasculitis
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Voxelotor Results in Hematologic Improvements for Sickle Cell Disease
Some of the favorable responses to voxelotor treatment and signs of hematologic response after voxelotor treatment included increased Hb levels, decreased reticulocyte percentage, and decreased total bilirubin.
Early Momentum for a Genomic Approach to Sickle Cell Disease Therapy
May 23rd 2022In a late-breaking presentation at ASGCT 2022, investigators shared preclinical results of a gene-editing strategy that seeks to reactivate developmentally silenced fetal hemoglobin (HbF, α2γ2) in order to replace defective sickle hemoglobin (HbS, α2βS2).
Phase 3 Data Show Isatuximab Offers Progression Free Survival for Multiple Myeloma
May 17th 2022Isatuximab in combination with carfilzomib and dexamethasone (Kd) demonstrate the longest median progression free survival (mPFS) on a proteasome inhibitor backbone in multiple myeloma patients who relapsed after a prior therapy, including lenalidomide.
Edward Chen, MD: Amassing Data on Sarcoidosis Prevalence in Rural and Suburban Areas in the US
May 17th 2022With this new decade-long cohort, Dr. Chen notes that researchers are "one step closer to a more accurate view of what sarcoidosis may be like in the United States", with has been suggested to be more prominent in African American communities.
New Study Supports the Reliability SCD Patient-Reports of VOC-Related Pain Crises
May 17th 2022Investigators who say that assessing vaso-occlusive crises solely through medical utilization doesn’t provide a full picture of the SCD experience because many VOCs are managed from home, are now backed by research.