Thrive with Pyruvate Kinase Deficiency

Volunteers needed to participate in a study investigating #fatigue. If you are an adult (aged 18 and above) currently living in the United States of America with a diagnosis of either #sicklecelldisease, #pyruvatekinasedeficiency or alpha/beta #thalassemia we would like to talk to you about your condition. We are conducting a research study that involves a single 45-minute interview, either online or by telephone. The purpose of this interview study is to understand your experience of living with sickle cell disease, pyruvate kinase or alpha/beta thalassemia. We will ask you about any fatigue you may experience. Taking part will help us to understand your experiences and may help other patients in the future. Taking part is entirely voluntary and your personal information would be kept confidential. You will be compensated for your time. If you are interested in taking part or would like more information, please contact the Clarivate study team at fatigue@clarivate.com. The study team are happy to answer any questions you may have. Clarivate is running the study on behalf of a company called Agios Pharmaceuticals. Tamara Schryver participates on a patient advisory panel for Agios Pharmaceuticals. #pkdeficiency #pyruvatekinasedeficiency #pkd #redbloodcelldisorders #hemolyticanemias #rareanemias #research #inheritedanemia #ironoverload #tired #rarediseases

Feelings of isolation and loneliness, often experienced by those with chronic diseases like hemolytic anemias, can lead to suicidal ideation. It's time to break the stigma of #suicide and start having conversations where people can show support for one another. If you don't feel like you can talk to your people, dial 988 in the U.S. or visit the International Association for Suicide Prevention. www.iasp.info. #StartTheConversation #suicideprevention #hemolyticanemias #pyruvatekinasedeficiency #sicklecellanemia #thalassemia #rarebutnotalone

The CDC is issuing an advisory alert to the public about increases in human #parvovirusB19 in the U.S. which is spread by respiratory droplets and can lead to serious health risks in people with chronic #hemolyticanemias like #pyruvatekinasedisease, #sicklecelldisease, or #thalassemia. Practice good hand hygiene and consider wearing a mask for prevention.😷 https://lnkd.in/gQS2Af6w #pkd #rareanemias #pkdeficiency

Not sure how to get involved in the PEAK Registry? Here's the cheat sheet link for sign-up and a cheat sheet! https://lnkd.in/gK7gSjEf #pyrvuatekinasedeficiency #pkdeficiency #PKD #rareanemia #peakregistry

What do the #redbloodcells look like in an adult with #pkdeficiency who has had her spleen removed? A recent case-study highlights the special features of #RBCs as they undergo #hemolysis and adapt to #loweroxygen, #lowerenergy environments of anemia. 🩸 https://ow.ly/PSJM50SREPO #pyruvatekinasedeficiency #echinocytes #spikeycells #hematology #rareanemias #blooddisease #blooddisorder #PKD #anemia

We are thrilled to pass along the preliminary results of Agios' Phase 3 ACTIVATE-Kid transfusion dependent trial as this may provide for another treatment option for children with #PKdeficiency in the future. 💊🚸 A total of 49 patients aged 1-18 years, 39 in the mitapivat arm, and 17 in the placebo arm: *28% of the patients in the mitapivat arm achieved a transfusion reduction response compared to 11.8% in the placebo arm, which was not statistically significant but clinically meaningful. *18.8% of the patients in the mitapivat arm became transfusion-free compared to 0 in the placebo arm *12.5% of the patients in the mitapivat arm achieved a normal hemoglobin response compared to 0 in the placebo arm *In the 32-week study, a similar proportion of patients in the mitapivat and placebo arm had adverse events and there was no discontinuation of study treatment due to adverse events *The safety profile of mitapivat in the pediatric study was similar to the transfused adults study For more details, visit: https://ow.ly/qXlS50SQOXX Disclosures: Thrive with PKD is not paid for promoting this information. Tamara Schryver is a member of Agios' Red Cell Revolution Advisory Council.

#Intheroomwhereithappened, Dr. Rachel Grace, Tamara Schryver, and Alejandra Watson discuss the patient and caregiver perspective of how the #pkdguidelines came together. Sponsored by Agios, catch this episode of the podcast Just Listen: Voices of PK Deficiency. https://ow.ly/zPEB50SNGIT #pkdeficiency #pyruvatekinasedeficiency #rareanemias #blooddisease #blooddisorder #pkd

When all that hard work and planning pays off, sponsors and donors come together, and people and families with #PKdeficiency are served, we call that #success! 🥳 Thank you to everyone for your efforts! Looking forward to #PKDconference2026! #pyruvatekinasedeficiency #PKDconnections #rareanemias #ironoverload #blooddisorder #blooddisease

#AgingwithPKD from the perspective of those living it! For those wondering what it's like, you'll want to make sure to check this out! #PKdeficiency #pyruvatekinasedeficiency #PKD #rareanemia #bridginglivesPKD

Today's patient and caregiver panel features members of the #pkdeficiency community who have participated in #PKD related research to further contribute to improving lives of those affected. Thank you! 👏 #pyruvatekinasedeficiency #bridginglivesPKD #rareanemias #livedexperience #research