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.

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Cilta-cel Improves Progression-Free Survival as Multiple Myeloma Therapy in Phase 3 Data
Cilta-cel Improves Progression-Free Survival as Multiple Myeloma Therapy in Phase 3 Data

January 27th 2023

The study population consists of patients who received 1-3 prior lines of therapy for relapsed and lenalidomide-refractory multiple myeloma.

FDA Expands Label of Sutimlimab-jome for Rare Disease to Include Adults Without Transfusion History
FDA Expands Label of Sutimlimab-jome for Rare Disease to Include Adults Without Transfusion History

January 26th 2023

Investigators Call for Education, Sickle Cell Disease-Modifying Therapies for Pregnant Women
Investigators Call for Education, Sickle Cell Disease-Modifying Therapies for Pregnant Women

January 16th 2023

Evan Dellon, MD, MPH: Assessing the Future of Dupilumab for EoE
Evan Dellon, MD, MPH: Assessing the Future of Dupilumab for EoE

January 13th 2023

FDA will not Take Action on Potential Erosive Esophagitis
FDA will not Take Action on Potential Erosive Esophagitis

January 5th 2023

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