Rare diseases are not actually that rare. Collectively they affect 500 million people around the world. Some, such as Huntington’s disease and sickle cell disease, affect many thousands, others only ...

Stargardt programs address ABCA4 payload constraints via dual-AAV platforms (VG801, AAVB-039) and explore first ...

Watch Toronto hospital looks to provide life-long care to sickle cell disease patients closer to home Video Online, on GlobalNews.ca ...

You Look Fine” is a groundbreaking new documentary, offering an intimate, first-person account of living with sickle cell disease.

“It can hit you out of nowhere,” she tells TODAY.com.

As Uganda rolls out mandatory nationwide screening of newborns for sickle cell disease this month, a gene therapy celebrated in the United States and Europe remains financially out of reach for most ...

CRISPR Therapeutics (NASDAQ:CRSP) executives highlighted progress across its oncology and autoimmune cell therapy pipeline during Citi’s Virtual Oncology Leadership Summit, emphasizing updates on its ...

Florida rapper Lil Poppa has died at 25. Fans are remembering him while talking openly about his battle with sickle cell ...

Rapper Lil Poppa, 25, has died, with unverified suicide reports circulating. His death raises concerns due to his battle with sickle cell anemia, which he previously discussed. The cause of death ...

Steffen Schmitz-Valckenberg, MD, is professor of ophthalmology and visual science and the Jon M. Huntsman Presidential Chair at John A. Moran Eye Center at the University of Utah in Salt Lake City, UT ...

PYRUKYND® (mitapivat) worldwide net revenues of $20.0 million in fourth quarter and $54.0 million for full yearAQVESME™ (mitapivat) for thalassemia now available in U.S. following FDA approvalCompany ...

Chlamydia pneumoniae is an intracellular bacterium that commonly causes respiratory infections such as pneumonia and sinusitis. It can persist in the body by evading immune defenses, leading to ...