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.

Latest News

Michael B. Gorin, MD, PhD | Image Credit: UCLA
Michael B. Gorin, MD, PhD: Interim TEASE-3 Trial Data in Stargardt Disease

January 10th 2024

Positive interim data from the TEASE-3 clinical trial show gildeuretinol slows the progression of Stargardt Disease for up to six years.

The Lupus Approach: CAR-T Begins the Jump from Blood Cancers to Autoimmune Disease
The Lupus Approach: CAR-T Begins the Jump from Blood Cancers to Autoimmune Disease

December 20th 2023

Etranacogene Dezaparvovec Significantly Improves Hemophilia B Past 2 Years
Etranacogene Dezaparvovec Significantly Improves Hemophilia B Past 2 Years

December 12th 2023

After Elevidys: DMD Advocacy Past First Gene Therapy Approval
After Elevidys: DMD Advocacy Past First Gene Therapy Approval

December 4th 2023

FDA Approves Nirogacestat Tablets for Rare Desmoid Tumors
FDA Approves Nirogacestat Tablets for Rare Desmoid Tumors

November 28th 2023

Video Interviews
Podcasts
Autoimmune Hepatitis at ACG 2023, with Gina Choi, MD
Latest CME Events & Activities

Expert Illustrations & Commentaries™: Targeting the Complement System to Optimize Treatment of Cold Agglutinin Disease

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Addressing Healthcare Inequities: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis

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BURST CME™: Taking ALS Management to the Next Level

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BURST Expert Illustrations & Commentaries™: A Closer Look at the Role of Complement Inhibitors in the Treatment of Paroxysmal Nocturnal Hemoglobinuria

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Oncology Briefings: How Do We Integrate New Data and Treatment Options to Optimize Outcomes for Patients with Hemolytic Anemias?

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Clinical ShowCase™: Taking Control of Paroxysmal Nocturnal Hemoglobinuria

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Cases and Conversations™: Enhancing Diagnosis and Management of Patients with Autoimmune Hemolytic Anemia

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Strategies to Manage Transfusional Iron Overload in Patients with ß-hemoglobinopathies: Optimizing Patient Outcomes

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Community Practice Connections™: Enhancing Diagnosis and Management of Patients with Autoimmune Hemolytic Anemia

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Let’s Get “Real”: Alpha-1 Antitrypsin Deficiency—Case-Based Perspectives on Managing Associated Emphysema

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Cases and Conversations™: Applying Guidelines to Practice for the Management of Paroxysmal Nocturnal Hemoglobinuria

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(CME) Community Practice Connections™: A Closer Look at Neurotrophic Keratitis—Ensuring Timely Diagnosis and Taking Early Action

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(COPE) Community Practice Connections™: A Closer Look at Neurotrophic Keratitis—Ensuring Timely Diagnosis and Taking Early Action

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Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management

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EGPA: Highlighting the Patient Journey to Improve the Differential Diagnosis and Accelerate the Initiation of Guideline-Based Care

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‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease

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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders

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Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders

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Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis

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Improving Outcomes in Autoimmune Hemolytic Anemias at the Intersection Between Hematology and Oncology Care

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Oncology Consultations®: Community and Academic Perspectives on PNH Management – How Do You Do It?

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Expert Illustrations & Commentaries™: Exploring the Role of Novel Agents for the Management of IgA Nephropathy

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