Browsing: rare

A drug called Tavneos used to treat rare autoimmune diseases will be pulled from the shelves in France, the country’s medicine watchdog said Wednesday, after it was linked to 20 deaths in Japan

 The Michigan health department on Wednesday confirmed a locally acquired case of eastern equine encephalitis, a rare mosquito-borne disease

The University of Texas at Arlington is creating a new approach to its graduate program in which doctoral students will train to become experts in a single rare disease while moving their research from the lab to real-world diagnosis, clinical trials and treatment

Shares of Ultragenyx Pharmaceutical (RARE) fell for the second consecutive day on Tuesday ahead of a closely watched U.S. Food and Drug Administration decision on its gene therapy slated for this weekend

N.C. (WITN) – An 11-year-old boy with a rare, terminal genetic condition went surfing for the first time this past weekend at Atlantic Beach

LEO Pharma, a global leader in medical dermatology delivering innovative solutions for skin disease, has entered into an agreement to acquire worldwide rights to dersimelagon from Japan-based Tanabe Pharma. Dersimelagon is an oral, once-daily small MC1R agonist being developed for erythropoietic protoporphyria (EPP) and X-linked proto-porphyria (XLP)

CNBC Cures is proudly under AstraZeneca Rare Disease, whose support enables our journalism to elevate stories that advance research, innovation and patient care in rare diseases

Shannon Knight, a brain and cognitive sciences PhD candidate and McGovern Institute researcher, focuses on developing a novel gene therapy. Stefanie Koperniak|Office of Graduate Education Publication Date: August 18, 2026

A conversation between Jen A. Levitt, MD, and Clinical Leader Executive Editor Abby Proch

Helen Sabin was told by specialists she had just five years to live when she was diagnosed with a rare lung condition in 2008