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
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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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From the Top Down: Fibrocell Studies Adults with Goal to Treat Pediatric Patients
February 22nd 2018In the early stages of the FCX-007 program, no children were included. Fibrocell has been testing the potential therapy in adults until enough successful data were collected to justify treating the pediatric population.
Orphan Drug Designation Granted to Pediatric Hepatitis B Therapy
February 22nd 2018ContraVir Pharmaceuticals, Inc. announced that the U.S. FDA has granted orphan drug designation to TXL, an investigational drug candidate for the potential treatment of chronic hepatitis B infection in a pediatric patient population.
Shire Submits sBLA for HAE Therapy to Include Pediatric Indication
The FDA has accepted a supplemental Biologics License Application (sBLA) for Shire’s Cinryze, a therapy that treats recurring swelling attacks in hereditary angioedema (HAE), to expand the already approved indication to include children 6 years and older.
FDA Confirms Soleno's Plan to Initiate Phase III Clinical Study to Treat PWS
Soleno Therapeutics has completed its End-of-Phase II meeting with the FDA regarding the biopharmaceutical company’s drug, Diazoxide Choline Controlled-Release (DCCR), a therapy used to treat patients with Prader-Willi syndrome (PWS).
GS010 Proves to be Safe and Tolerable During Phase I/II Trials in LHON Patients
GenSight Biologics has published successful results in the Journal of the American Academy of Ophthalmology from their Phase I/II clinical trial and long-term follow-up of GS010, a therapy used to treat patients with Leber Hereditary Optic Neuropathy (LHON).
American Lung Association to Host Upcoming Expo and Webinar
The American Lung Association will host two events this spring: the 2018 Greater Philadelphia LUNG FORCE Expo and a webinar for sarcoidosis patients focusing on improving health outcomes and support programs.
Takeda Releases New Data Proving Effectiveness of Vedolizumab in UC and Crohn's Disease
February 20th 2018Takeda released new real-world data evaluating the comparative effectiveness of Entyvio (vedolizumab) and TNFα-antagonist in patients with moderately to severely active ulcerative colitis or Crohn’s disease.
FDA Commissioner Releases Statement Highlighting Plans for Rare Disease Research
February 14th 2018Late last night, U.S. FDA Commissioner Scott Gottlieb released a statement regarding the Administration’s request for new FDA funding to “promote innovation and broaden patient access through competition.”
Researcher at the Feinstein Institute Receives Grants from DOD and NIH to Study HHT
A researcher at the Feinstein Institute for Medical Research has been awarded two grants for clinical trials on existing FDA-approved drugs that can be indicated to treat hereditary hemorrhagic telangiectasia.