Agios Pharmaceuticals is discontinuing development of its oral therapy candidate, tebapivat, for people with sickle cell disease (SCD). The decision follows results from a small Phase 2 clinical trial (NCT06924970) showing that while tebapivat — an activator of the pyruvate kinase (PK) enzyme — reduced measures of red…
Discussion
As both a physician and someone living with sickle cell disease (SCD), I spend a great deal of time reading SCD-related medical literature. Recently, while reviewing several publications, both old and recent, on vaso-occlusive crises, I noticed recurring statements that made me pause: Acute sickle cell pain and vaso-occlusive…
Discussion
In the U.S., doctors who care for people with sickle cell disease (SCD) often don’t have a clear understanding of what motivates patients to participate in clinical trials, according to a survey-based study called LISTEN. Based on the findings, researchers offered a range of suggestions for how doctors can…
Discussion
Years ago, I mistook a pulmonary embolism for what I thought was a minor sickle cell disease vaso-occlusive pain crisis, a mistake that could easily have proved fatal. I promised myself afterward that I would never trivialize my symptoms again. As it turns out, that’s easier said than done.
Becoming educated and informed about sickle cell disease is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your sickle cell disease journey.
A stem cell transplant is the only potential cure for sickle cell disease, but there are a variety of other treatment approaches. Here you can find more information about current treatments as well as experimental therapies in the pipeline.
Get regular updates to your inbox.
