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 5th 2024
Katie Abouzahr, MD, explains the transformative potential of nipocalimab for the treatment of patients with autoantibody-driven conditions.
Addressing Healthcare Inequities: Tailoring Management Plans to Address Healthcare Disparities in Cystic Fibrosis
View More
BURST Expert Illustrations & Commentaries™: A Closer Look at the Role of Complement Inhibitors in the Treatment of Paroxysmal Nocturnal Hemoglobinuria
View More
Oncology Briefings: How Do We Integrate New Data and Treatment Options to Optimize Outcomes for Patients with Hemolytic Anemias?
View More
Clinical ShowCase™: Taking Control of Paroxysmal Nocturnal Hemoglobinuria
View More
Cases and Conversations™: Enhancing Diagnosis and Management of Patients with Autoimmune Hemolytic Anemia
View More
Strategies to Manage Transfusional Iron Overload in Patients with ß-hemoglobinopathies: Optimizing Patient Outcomes
View More
Community Practice Connections™: Enhancing Diagnosis and Management of Patients with Autoimmune Hemolytic Anemia
View More
Let’s Get “Real”: Alpha-1 Antitrypsin Deficiency—Case-Based Perspectives on Managing Associated Emphysema
View More
(CME) Community Practice Connections™: A Closer Look at Neurotrophic Keratitis—Ensuring Timely Diagnosis and Taking Early Action
View More
Cases and Conversations™: Applying Guidelines to Practice for the Management of Paroxysmal Nocturnal Hemoglobinuria
View More
(COPE) Community Practice Connections™: A Closer Look at Neurotrophic Keratitis—Ensuring Timely Diagnosis and Taking Early Action
View More
Advances In™ Generalized Myasthenia Gravis: Improving Patient Outcomes Through Early Diagnosis and Management
View More
EGPA: Highlighting the Patient Journey to Improve the Differential Diagnosis and Accelerate the Initiation of Guideline-Based Care
View More
Burst Expert Illustrations & Commentary™ : Visualizing the Role of the Complement Proteins in Neurologic Disorders
View More
Burst Expert Illustrations & Commentary™: Visualizing the Role of the Complement Pathway in Neurological Disorders
View More
‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease
View More
Burst Expert Illustrations & Commentary™: Visualizing the Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
View More
Clinical ShowCase™ in ALS: Addressing Diagnostic Delays, Evolving Therapies, and Multidisciplinary Care
View More
Improving Outcomes in Autoimmune Hemolytic Anemias at the Intersection Between Hematology and Oncology Care
View More
Oncology Consultations®: Community and Academic Perspectives on PNH Management – How Do You Do It?
View More
Expert Illustrations & Commentaries™: Exploring the Role of Novel Agents for the Management of IgA Nephropathy
View More
IFFGD Will Participate in Rare Disease Day to Support Rare Digestive Disease Patients
The International Foundation for Functional Gastrointestinal Disorders will participate in Rare Disease Day on February 28, with efforts to support patients affected by a rare digestive disease globally.
Alzheimer's Treatment Prevents and Improves Neurological Damage on Mouse Model
A collaborative research effort between the National Institute on Aging (NIA), the National Institutes of Health (NIH), and ChromaDex has found Niagen effective in preventing neurological damage and improving cognitive and physical function in a mouse model of Alzheimer’s disease.
Amplify Hope Study Educates Families on Crowdfunding for Genetic Testing
The Amplify Hope Study, sponsored by the Rare Genomics Institute, analyzed the effects of educating families affected by rare disease on how to effectively crowdfund to pay for necessary genetic testing.
CHLA Takes On Trial of Potential Stem Cell Procedure in Treating HLHS
Children’s Hospital Los Angeles (CHLA) has announced that it will be participating in a new clinical trial with the hopes of improving quality of life (QOL) outcomes for children with hypoplastic left heart syndrome (HLHS), a congenital heart defect in which the left ventricle is severely underdeveloped.
BioPontis Alliance and Massachusetts General Hospital Partner for HD
February 2nd 2018Yesterday, nonprofit organization BioPontis Alliance and Massachusetts General Hospital, the largest hospital-based research program in the United States, announced an agreement to target new treatments for Huntington’s disease.