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Rare Diseases

Latest News & Journal Articles View All

Examining Physician Legislators in the US Congress 2011-2020

Southern Medical Journal May 23, 2022
Case of Monkeypox Confirmed in Massachusetts

WebMD Health News May 19, 2022
Alert

FDA Approves Oral Form of ALS Drug Edaravone

FDA Approvals May 13, 2022
FDA Does Not Approve TKI Surufatinib for Neuroendocrine Tumors

News Alerts May 2, 2022
Alert

Topical Treatment for EB Recommended for Approval in the EU

International Approvals April 27, 2022
New Ebola Outbreak Declared in Democratic Republic of Congo

WebMD Health News April 25, 2022
Early Promise for New Agent in Congenital Hyperinsulinism in Kids

Medscape Medical News April 21, 2022
AACR 2022

More Drugs Tested in Pediatric Cancer: RACE for Children Act

Medscape Medical News April 11, 2022

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Perspective View All

Cure, Prevent, or Manage All Disease: The Chan Zuckerberg Initiative's Bold Plan Is Already Working

Expert Interview April 28, 2022

Dr Eric Topol sits down with Mark Zuckerberg and Priscilla Chan to talk about the next phase of their ambitious Chan Zuckerberg Initiative.
When Physicians Are the Plaintiffs

Blog February 25, 2022
American College of Cardiology & Medscape

Navigating Prior Authorization and Costs for HFrEF Drugs

Commentary May 11, 2021
Why Biliary Atresia Demands Our Respect

Commentary August 19, 2020

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Case Challenges View All

An Incarcerated 24-Year-Old With Dyspnea, Fatigue, and Nausea

Case Challenge March 18, 2022

A 24-year-old man presents with symptomatic anemia. His primary symptoms are exertional dyspnea, generalized fatigue, and some mild chronic nausea. What's the diagnosis?
17-Year-Old With Hair Loss, Dysmenorrhea, Thrush, and Diarrhea

Clinical Case March 15, 2022
A 16-Year-Old Girl With Full-Body Rash, Dyspnea, and Swelling

Case Challenge January 19, 2022
A Nonverbal 33-Year-Old Woman With Intellectual Impairment

Case Challenge October 20, 2021

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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