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.
September 26th 2023
Announced by Ionis Pharmaceuticals, topline results show olezarsen met the study's primary endpoint with a statistically significant reduction in triglyceride levels versus placebo.
Community Practice Connections™: Chronic Fibrosing ILDs With Progressive Phenotype–Are You Aware of the Latest Data on Diagnosis and Emerging Treatments?
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Free COPE CE at AAOPT
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Taking Pompe Disease Management to the Next Level: Optimizing Clinical Assessments and Treatment Decision-Making
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C.R.U. Eye Symposium (Current, Relevant, Useful)
11/10/2023-11/12/2023
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Addressing Healthcare Inequities in Cardiac Amyloidosis: Interventions Aimed at Improving Care for All Patients
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Addressing Healthcare Inequities: Overcoming Challenges in the Equitable Management of Idiopathic Pulmonary Fibrosis
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Clinical Consultations™: Raising Awareness and Optimizing Treatment in Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
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Patient, Provider, and Caregiver Connection™: Incorporating the Patient Journey into HS Diagnosis and Management Practices
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Community Practice Connections: Optimizing Recognition and Management of Acute Hepatic Porphyria
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Addressing Healthcare Inequities: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis
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Advances In™ Desmoid Tumors: Recognizing Disease Burden, Pathophysiology, and Targeted Treatments in Development
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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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"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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Community Practice Connections™: 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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Clinical ShowCase™: Taking Control 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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Cases and Conversations™: Enhancing Diagnosis and Management of Patients with Autoimmune Hemolytic Anemia
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Medical Crossfire®: 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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Improving Outcomes in Autoimmune Hemolytic Anemias at the Intersection Between Hematology and Oncology Care
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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.
Robert Hariri, MD: Natural Killer Cell Therapy Shows Remarkable Potential in Treating Relapsed AML
May 31st 2023Celularity's clinical trial of CYNK-001 for relapsed and refractory AML showed excellent tolerability, significant graft-versus-leukemia effects, and potential for flexible therapeutic development.