Home2020/06 June 27, 2020 -

https://mysicklefamilyllc.com/wp-content/uploads/2020/06/shutterstock_88062898.jpg

Mitapivat (AG-348) safely increased the amount of hemoglobin and changed the levels of biomarkers of red blood damage and energy production in adults with sickle cell disease (SCD), early Phase 1 trial data show. “These data build on our six years of clinical experience with this mechanism and establish proof-of-concept for mitapivat as a potential novel approach for the treatment of sickle cell disease,” Chris Bowden, MD, chief medical officer at Agios Pharmaceuticals, mitapivat’s developer,...

https://mysicklefamilyllc.com/wp-content/uploads/2020/06/shutterstock_273577295-1400x480@2x-1280x480.jpg

We celebrated World Blood Donor Day on June 14. It reminded me that blood donors are a heavily relied upon and greatly anonymous source of support all over the world. If blood donations did not exist, medicine as we know it would be so different; the whole system would collapse. The purpose of this column is to acknowledge and thank blood donors everywhere. Irrespective of the reason that you donate, the fact that you have...

https://mysicklefamilyllc.com/wp-content/uploads/2020/06/shutterstock_365583293_zpsl1o0mej7.jpg

The first patient with severe sickle cell disease (SCD) who received a single infusion of CTX001, an experimental gene-editing cell therapy, remains free of vaso-occlusive crises (VOCs) nine months after treatment, a Phase 1/2 clinical trial shows. Findings from the trial, “(S280) Initial Safety and Efficacy Results With a Single Dose of Autologous CRISPR-CAS9 Modified CD34+ Hematopoietic Stem and Progenitor Cells in Transfusion-dependent B-thalassemia and Sickle Cell disease,” were presented at the recent European Hematology...

https://mysicklefamilyllc.com/wp-content/uploads/2020/06/shutterstock_186943337-1280x720.jpg

The Alexion Charitable Foundation has awarded $1.1 million in grants to programs that support those with rare diseases during the COVID-19 pandemic, the organization recently announced. The grants will support activities that align with the foundation’s Rare Belonging focus, a set of funding priorities aimed at improving the daily lives of those with rare diseases. These priorities include mental health counseling, education and job skills, transportation and accommodation, as well as other critical needs. Global...

https://mysicklefamilyllc.com/wp-content/uploads/2020/06/shutterstock_344201303-e1553120302266.jpg

Whenever I meet new people and tell them I have sickle cell disease (and explain what it is), they often ask how they can help. June 19 is World Sickle Cell Awareness Day, so I thought it appropriate to discuss ways that you can help the sickle cell community, including friends who may have the disease. Here’s how to help Because many of the affects of sickle cell disease are internal, the sad reality is...

https://mysicklefamilyllc.com/wp-content/uploads/2020/06/Hertz-Nazaire_Profile-Pic.jpg

Supporters are poised to mark World Sickle Cell Day, observed each June 19 to raise awareness about the red blood cell disorder, and to raise funds to fight it. Across the globe, supporters will participate in activities such as wearing red for sickle cell disease (SCD), sponsoring a walk or run, shaving or dying their hair, and sharing patient stories with local media outlets. Organized by the U.K.-based Sickle Cell Society, the event also is...

https://mysicklefamilyllc.com/wp-content/uploads/2020/06/shutterstock_243828643.jpg

June 19 is World Sickle Cell Day, and I have been reflecting a lot on my life with sickle cell. From a young age, I was always encouraged to keep my diagnosis hush-hush. People would only know I had sickle cell if they needed to know. In my household, my diagnosis was always viewed as a setback. My parents did not want me to be discriminated against due to my health — they’ve seen that...