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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FDA Approves First Adjunctive Treatment for TSC-Associated Seizures
April 11th 2018This morning, it was announced that everolimus (Afinitor DISPERZ) has become the first therapy to be specifically approved by the U.S. FDA to treat patients aged 2 years and older with tuberous sclerosis complex (TSC)-associated partial-onset seizures.
HBO Documentary Shines Spotlight on World's Most Famous Case of Acromegaly: Andre the Giant
April 11th 2018André the Giant, examines the life and work history of the WWE Hall of Famer. Unbeknownst to many, his size was the result of gigantism, a disease caused by excess growth hormone. The condition led to his eventual diagnosis with acromegaly, a rare endocrine disorder.
Orphan Drug Designation Granted to RP5063 for Second Pulmonary Indication
April 10th 2018Early this morning, Reviva Therapeutics announced that the U.S. Food and Drug Administration has granted orphan drug designation to its clinical stage drug candidate RP5063, in development for the treatment of idiopathic pulmonary fibrosis.
IPF KOL Discusses Current and Emerging Therapies
April 9th 2018At the most recent American Lung Association LUNGFORCE expo, RDR sat down with Albert Rizzo, M.D. of the Christiana Care Pulmonary and Critical Care Medicine Section to discuss previous, current and emerging therapies for idiopathic pulmonary fibrosis.
Abortion Drug Serves Secondary Purpose to Cushing Community
April 9th 2018When taken early in a pregnancy, mifepristone can end a pregnancy that is less than 49 days along. Another notable trait of the drug, though, is its ability to control the high blood sugar in patients with Cushing syndrome who have type 2 diabetes and have either failed surgery or are not candidates for it.
Five Facts About: Myeloproliferative Neoplams (MPNs) [Infographic]
April 9th 2018Rare Disease Report tries to keep its audience at the forefront with the latest news from the hematological and oncological spaces. This new infographic provides 5 little-known facts about MPNs that might not necessarily be covered in news articles from RDR or other media outlets.
Orphanet Publishes Clinical Management Guidelines for NPC
April 7th 2018Tarekegn Geberhiwot, MD, of the Institute of Metabolism and Systems Research and colleagues had an article published in the Orphanet Journal of Rare Diseases, outlining the development of clinical management recommendations for Niemann-Pick disease type C.
New Research Exhibits Benefits of Nivolumab Plus Ipilimumab in Renal Cell Carcinoma
April 6th 2018Treatment with nivolumab, followed by ipilumab, produced significantly higher overall survival and objective response rates than sunitinib alone among intermediate- and poor-risk patients with previously untreated advanced renal-cell carcinoma.
Fragile X Imaging Study Results Show Differences in Infant Brains
April 5th 2018Using MRIs and computer models, researchers from the University of North Carolina School of Medicine were able to prove that babies who develop the fragile X syndrome have less white matter circuitry than infants who did not.