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 25th 2024
On October 25, 2024, Kind Pharmaceutical announced receipt of FDA Orphan Drug Designation for AND017 in the treatment of sickle cell disease.
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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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.
Advocacy: The Solution to Sickle Cell Disease
December 22nd 2021The American Society of Hematology debuted its Health Equity Rounds series, which are interactive, case-based discussions with an interdisciplinary panel to include hematologists, health equity/public health experts, patients, and patient advocates.