Rare Disease Report®

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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.

Latest News

Ophthalmoscopy Insufficient to Detect Vitreoretinopathy in CTNNB1 Syndrome | Image Credit: LinkedIn
Ophthalmoscopy Insufficient to Detect Vitreoretinopathy in CTNNB1 Syndrome

August 16th 2024

Patient Reported Outcomes Favor Concizumab Prophylaxis in Hemophilia A or B | Image Credit: Alfred Health
Patient Reported Outcomes Favor Concizumab Prophylaxis in Hemophilia A or B

August 15th 2024

FDA Approves Axatilimab for Chronic Graft-Versus-Host Disease| Image Credit: US Food and Drug Administration
FDA Approves Axatilimab for Chronic Graft-Versus-Host Disease

August 14th 2024

Ravulizumab Boosts Activity Levels, Quality of Life in Patients with PNH | Image Credit: Mayo Clinic
Ravulizumab Boosts Activity Levels, Quality of Life in Patients with PNH

August 12th 2024

Ocugen, Inc. Announces FDA Approval of Expanded Access Program for Patients with Retinitis Pigmentosa | Image Credit: Duke University Eye Center
FDA Approves OCU400 Expanded Access Program for Retinitis Pigmentosa

August 5th 2024

Latest CME Events & Activities

Expert Illustrations & Commentaries™: Exploring the Role of Novel Agents for the Management of IgA Nephropathy

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‘REEL’ Time Patient Counseling™: Navigating the Complex Journey of Diagnosing and Managing Fabry Disease

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Clinical ShowCase™: Forming a Personalized Treatment Plan for a Patient With ANCA-Associated Vasculitis

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