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Rare Diseases

Latest News & Journal Articles View All

Alert

FDA Approves Imaging Drug for Detecting Ovarian Cancer Lesions

FDA Approvals November 29, 2021
Interpreting Cortisol and SSTs to Prevent Misdiagnosis of AI

Journal of the Endocrine Society November 18, 2021
EMA Panel Backs Uplizna for Neuromyelitis Optica

International Approvals November 15, 2021
Alert

Miltefosine Granted Orphan Drug Approval for Invasive Candidiasis

FDA Approvals November 12, 2021
KidneyWeek 2021

Lumasiran Effects in Rare Disease Seen Despite Kidney Status

Medscape Medical News November 9, 2021
X-Linked Hypophosphatemia Exacts Early, High Toll on Musculoskeletal System

Reuters Health Information November 2, 2021
Cervical Cancer Mortality Stagnates Despite Screening

Medscape Medical News October 28, 2021
ECTRIMS 2021

Which Agent Best for Neuromyelitis Optica?

Medscape Medical News October 26, 2021

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Perspective View All

American College of Cardiology & Medscape

Navigating Prior Authorization and Costs for HFrEF Drugs

Commentary May 11, 2021

Online tools and decision aids can help clinicians and patients work through the barriers that have slowed the use of new drugs such as sacubitril-valsartan.
Why Biliary Atresia Demands Our Respect

Commentary August 19, 2020
Maybe Memorizing the Krebs Cycle Was Worthwhile After All

Expert Interview February 6, 2020
Confessions of a 'High-Yield' Doctor

Commentary November 20, 2019

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Case Challenges View All

A Nonverbal 33-Year-Old Woman With Intellectual Impairment

Case Challenge October 20, 2021

A 33-year-old woman is seen with her caregivers. She has profound intellectual impairment and is thought to have the mental capacity of an 18-month-old child. Do you know what condition she has?
My Strangest Case: An Infant With Lumps in Her Belly

Case Challenge June 10, 2020
A 25-Year-Old Woman With a Droopy Eyelid and Double Vision

Case Challenge March 20, 2020
A 14-Year-Old Boy With Unexplained, Recurring Fever

Case Challenge June 17, 2019

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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