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 23rd 2024
Abeona's pz-cel is up for indicated use to treat patients with recessive dystrophic epidermolysis bullosa.
Addressing Healthcare Inequities: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis
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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 Proteins in Neurologic Disorders
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Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders
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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 Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
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Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care
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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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Debajyoti Ghosh, PhD: Potential Benefits of Treating Hereditary Angioedema Early Symptoms
February 27th 2023In a presentation at the AAAAI 2023 Annual Meeting, new data suggested that treating early symptoms may be a viable strategy for preventing acute attacks in patients with hereditary angioedema (HAE).
Novel Iron Supplement Effective in Treating Iron Deficiency Anemia in Children
February 21st 2023Phase 2 trial shows iron hydroxide adipate tartrate (IHAT) is non-inferior to ferrous sulfate (FeSO4) in correcting iron deficiency and hemoglobin response, with a lower incidence of moderate-severe diarrhea.
Velmanase alfa-tycv: First FDA-Approval for Non-CNS Manifestations of Alpha-Mannosidosis
February 17th 2023Velmanase alfa-tycv (Lamzede) is approved as the first and only enzyme replacement therapy for non-central nervous system manifestations of alpha-mannosidosis, an extremely rare, progressive, lysosomal storage disorder.
Weekly Efanesoctocog Alfa Improves Bleeding Prevention, Patient Health in Hemophilia A
February 15th 2023Once-weekly efanesoctocog alfa is a safe and effective treatment for patients with severe hemophilia A, providing better outcomes than current treatment options by significantly reducing bleeding rates and improving physical health, pain, and joint health.
New Data Show Severe Maternal Morbidity is a Threat to People with Sickle Cell Disease
February 2nd 2023"Despite advances in SCD management and high-risk pregnancy care, at the national level, outcomes in this population have not improved since the last NIS analysis of data from 1999 to 2008," investigators write.
Investigators Call for Education, Sickle Cell Disease-Modifying Therapies for Pregnant Women
January 16th 2023With public health efforts and therapeutic advancements the prognosis for this rare condition has progressed, but longer life expectancy has highlighted a multitude of areas that need attention in this population.
Phase 3 Data Support Myelodysplastic Syndromes Treatment as Potential First-in-Class Therapy
January 4th 2023Promising results from the IMerge phase 3 clinical trial met the primary and secondary endpoints in the investigation of imetelstat, a novel telomerase inhibitor developed for hematologic malignancies.
Neutrophil, Platelet Engraftment is Successful with Briquilimab in Patients with Sickle Cell Disease
January 3rd 2023The targeted patient population included those with sickle cell disease or beta thalassemia who were considered at high risk for complications, or otherwise ineligible for standard myeloablative hematopoietic stem cell transplant.