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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Parents Raise Money for Rare Disease Research through the Million Dollar Bike Ride at Penn Orphan Di
April 4th 2018Parents of children with the rare disease Congenital Muscular Dystrophy are spending the next seven weeks preparing for the 5th Annual Million Dollar Bike Ride, to be held in Philadelphia on May 20th.
Blincyto Approval Expanded for Acute Lymphoblastic Leukemia Subgroup
March 29th 2018The U.S. Food and Drug Administration (FDA) has granted accelerated approval to blinatumomab (Blincyto) for the treatment of adults and children with B-cell precursor ALL who are in remission, but still have minimal residual disease.
Pfizer Drug Reduces Mortality and Hospitalizations in Transthyretin Cardiomyopathy Patients
March 29th 2018In the ATTR-ACT study, tafamidis exhibited a statistically significant reduction in all-cause mortality and frequency of cardiovascular-related hospitalizations in transthyretin cardiomyopathy patients.
First Patient Treated in Phase 2 PREVENT Study of Exendin 9-39 for Post-Bariatric Hypoglycemia
Eiger announced first patient treated in the PREVENT study, a phase 2, multicenter study of subcutaneous (SC) exendin 9-39 in post-bariatric surgical patients who experience PBH.