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Rare Diseases

Latest News & Journal Articles View All

Phenotypic Shift, Varied Vasculopathy Noted in IPAH

Medscape Medical News July 26, 2024
Alert

Two Soliris Biosimilars Approved for PNH in the United States

Medscape Medical News July 24, 2024
Boy Lives in Permanent Shade to Survive Dangerous Sunlight

Reuters Health Information July 18, 2024
Alert

New Drugs Turned Down by EU Safety Assessor

Medscape Medical News June 28, 2024
EHA 2024

Hemophilia: Marstacimab Sustains Long-Term Bleeding Decrease

MDedge News June 28, 2024
PNS 2024

Promising Agent Significantly Improves Disability in GBS

Medscape Medical News June 27, 2024
Virtual Collaboration Boosts Rare Cancer Care in Europe

Medscape Medical News June 27, 2024
Savara's Rare Lung Disease Therapy Succeeds in Late-stage Study

Reuters Health Information June 26, 2024

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Perspective View All

C3 Glomerulopathy: Not Just for Nephrologists

Commentary May 20, 2024

Dr Gerald Appel discusses the importance of complement blockers in emerging treatments for C3 glomerulopathy and other diseases.
C3 Glomerulopathy Diagnosis

Commentary May 9, 2024
Q&A: PIK3CA-Related Overgrowth Spectrum

Commentary April 29, 2024
Hereditary Amyloidosis: 5 Things to Know

Commentary April 29, 2024

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Case Challenges View All

Fact or Fiction: Complement 3 Glomerulopathy

Interactive Quiz April 5, 2024

Can you separate fact from fiction when it comes to C3 glomerulopathy? Test your knowledge with this quiz.
A 41-Year-Old on Sildenafil With a Headache While Sleeping

Case Challenge August 22, 2022
A Woman With Back, Chest Pain After Eating Wings at a Restaurant

Case Challenge May 31, 2022
An Incarcerated 24-Year-Old With Dyspnea, Fatigue, and Nausea

Case Challenge March 18, 2022

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Medscape Editorial Collaborations

Conditions in Focus

Advisory Board

Stephanie L. Austin, MS, MA

Priya S. Kishnani, MD

Marshall L. Summar, MD

Editor

Amy Nadel

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