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

Elucidating the Role of Type I Collagen Mutations on Respiratory Function in Osteogenesis Imperfecta
Elucidating the Role of Type I Collagen Mutations on Respiratory Function in Osteogenesis Imperfecta

September 17th 2024

Better known as brittle bone disease, osteogenesis imperfecta can cause severe respiratory distress at birth and longterm effect to respiratory function. Morello shares his team's research into the cause of this effect.

Nanoscope Reports Positive End-of-Phase 2 Meeting for MCO-010 Gene Therapy| Image Credit: US Food and Drug Administration
Nanoscope Reports Positive End-of-Phase 2 Meeting for MCO-010 Gene Therapy

September 13th 2024

FDA Grants Breakthrough Therapy Designation to Plozasiran for FCS | Image Credit: FDA
FDA Grants Breakthrough Therapy Designation to Plozasiran for FCS

September 12th 2024

ATSN-101 Gene Therapy Reports Positive Data in Phase 1/2 Trial | Image Credit: LinkedIn
ATSN-101 Gene Therapy Reports Positive Data in Phase 1/2 Trial

September 10th 2024

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

August 16th 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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