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.
December 4th 2023
For This Year in Medicine, CGTLive looks back at a landmark first approval, the new treatment opportunities it has provided, and the unmet needs in the field it has brought to light.
October 20th 2023
Patient, Provider, and Caregiver Connection™: Incorporating the Patient Journey into HS Diagnosis and Management Practices
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Addressing Healthcare Inequities: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis
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Community Practice Connections: Optimizing Recognition and Management of Acute Hepatic Porphyria
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Patient and Provider Connection™: Aligning Evidence-Based Management Strategies with Quality-of-Life Considerations to Optimize Care in Patients with HAE
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Transfusion-Related Iron Overload–Evidence-Based Approaches to Improve Chelation Outcomes
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Advances In™ Desmoid Tumors: Recognizing Disease Burden, Pathophysiology, and Targeted Treatments in Development
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"D" is for Diagnosis: Deciphering Uncommon Hematologic Disorders Through the Eyes of a Detective
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Addressing Healthcare Inequities™: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis
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Transforming How We Treat Sickle Cell Disease: Holistic Approaches to Addressing Clinical Challenges Across the Patient’s Lifespan
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Advances in™ Congenital Adrenal Hyperplasia: Investigating the Impact of Emerging Agents
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Expert Illustrations & Commentaries™: Targeting the Complement System to Optimize Treatment of Cold Agglutinin Disease
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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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(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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Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management
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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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Phase 4 Trial Shows Positive Results for Biweekly Dosing of Xembify in Primary Immunodeficiencies
July 24th 2023Investigators aimed to support extending the US FDA labeling of Xembify to include the biweekly dosing option, therefore, providing greater flexibility and convenience for patients managing their immunodeficiencies.
Roctavian Receives Approval by the FDA for Severe Hemophilia A
June 29th 2023The approval is supported by positive results from the largest and longest phase 3 study for gene therapy in hemophilia, showing a 52% reduction in annualized bleeding rate compared with baseline rates on routine prophylaxis.
Ambroxol Repurposing Improves Biochemical Markers in Gaucher Disease
June 28th 2023Significant improvements seen in hematologic parameters, visceral volumes, and plasma biomarkers in patients with Gaucher disease, suggesting its potential as an alternative therapy, particularly for those who cannot access enzyme replacement therapy.
Improving Outcomes for Patients with Sickle Cell Disease Amid Racial Disparities
June 20th 2023Among other associations, those in educational attainment, employment status, mental health, and pain episode frequency and severity highlight the need for comprehensive care that considers the psychological well-being of individuals with SCD.