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.
Improving Outcomes in Autoimmune Hemolytic Anemias at the Intersection Between Hematology and Oncology Care
View More
Oncology Briefings: How Do We Integrate New Data and Treatment Options to Optimize Outcomes for Patients with Hemolytic Anemias?
View More
Clinical ShowCase™: Taking Control of Paroxysmal Nocturnal Hemoglobinuria
View More
Cases and Conversations™: Enhancing Diagnosis and Management of Patients with Autoimmune Hemolytic Anemia
View More
Strategies to Manage Transfusional Iron Overload in Patients with ß-hemoglobinopathies: Optimizing Patient Outcomes
View More
Community Practice Connections™: Enhancing Diagnosis and Management of Patients with Autoimmune Hemolytic Anemia
View More
Oncology Consultations®: Community and Academic Perspectives on PNH Management – How Do You Do It?
View More
Let’s Get “Real”: Alpha-1 Antitrypsin Deficiency—Case-Based Perspectives on Managing Associated Emphysema
View More
(CME) Community Practice Connections™: A Closer Look at Neurotrophic Keratitis—Ensuring Timely Diagnosis and Taking Early Action
View More
Cases and Conversations™: Applying Guidelines to Practice for the Management of Paroxysmal Nocturnal Hemoglobinuria
View More
(COPE) Community Practice Connections™: A Closer Look at Neurotrophic Keratitis—Ensuring Timely Diagnosis and Taking Early Action
View More
Expert Illustrations & Commentaries™: Exploring the Role of Novel Agents for the Management of IgA Nephropathy
View More
Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management
View More
EGPA: Highlighting the Patient Journey to Improve the Differential Diagnosis and Accelerate the Initiation of Guideline-Based Care
View More
‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease
View More
Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
View More
Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders
View More
Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders
View More
Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care
View More