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.
March 8th 2024
With drug breakthroughs reaching epidermolysis bullosa, Paller considers what may come next for other rare skin diseases.
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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(CME) Community Practice Connections™: A Closer Look at Neurotrophic Keratitis—Ensuring Timely Diagnosis and Taking Early Action
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Cases and Conversations™: Applying Guidelines to Practice for the Management of Paroxysmal Nocturnal Hemoglobinuria
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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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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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‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease
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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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How Raising Awareness is Changing the Myasthenia Gravis Landscape
March 20th 2018In this video, Beth Stein, M.D. and myasthenia gravis patient Anaya Mitchell explain how raising awareness is changing the landscape for patients with myasthenia gravis and leading to medical breakthroughs for the patient population.
Study Reveals Commonality of Rare Neuropathy in Korean Population
March 16th 2018Results from a study published in the Orphanet Journal of Rare Diseases this week suggest that the hereditary neuropathy with liability to pressure palsies, which has been assumed to be rare, might not be uncommon in the Korean population.
Q&A with Hartmann Wellhoefer, Shire's VP and Head of Medical Affairs; Rare Diseases & Internal Medic
Hartmann Wellhoefer, M.D., head of medical affairs at Shire, recently sat down with Rare Disease Report to discuss the company's recent work for the rare disease community, particularly lysosomal storage diseases.