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.
March 8th 2024
With drug breakthroughs reaching epidermolysis bullosa, Paller considers what may come next for other rare skin diseases.
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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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 Pathway in Neurological Disorders
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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 Implications of Anti-Complement Therapies on Generalized Myasthenia Gravis
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‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease
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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 Allows Fibrocell to Initiate Pediatric Enrollment in Epidermolysis Bullosa Trial
January 30th 2018This morning, Fibrocell Science, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the company allowance to begin enrollment in the Phase 2 portion of its Phase 1/2 clinical trial of FCX-007.
FIREFISH Study Shows Clinical Benefit of RG7916 in Infants With SMA
PTC Therapeutics has announced that Part 1 of the FIREFISH study has shown that RG7916, a Type 1 spinal muscular atrophy (SMA) drug, to be safe and well-tolerated in infants and babies with Type 1 SMA.
Financial Coverage for Rare Disease Treatments Inconsistent in British Columbia
The Canadian Agency for Drugs and Technologies in Health organization’s Common Drug Review protocol often leaves rare disease patients in British Columbia without governmental payment coverage for their expensive treatments.
FDA Accepts IND and Grants Fast Track Status to Sanfilippo Drug
January 23rd 2018This morning, Sobi, announced that the FDA has issued a Study may proceed letter for the first study in humans, thereby accepting the investigational new drug application for sobi003, and the regulatory agency granted Fast Track status to the product candidate.