Home2020/05 May 14, 2020 -


Agios Pharmaceuticals announced that, despite the current COVID-19 outbreak, it remains on track to decide by mid-year next steps for mitapivat (AG-348) as a potential treatment of sickle cell disease. Mitapivat is in an ongoing proof-of-concept Phase 2 trial in up to 25 patients with stable SCD, but further enrollment has been suspended due to the pandemic. “We moved quickly to reduce the risk of our team’s and communities’ exposure to the virus and took action to enable uninterrupted access to...


The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is affecting people with rare diseases, their families, and caregivers. Survey questions cover a patient’s physical and mental health, supply of treatments, and access to healthcare, among other disease-related issues and those brought by the pandemic. Developed and led by the RDCRN Data Management and Coordinating Center at Cincinnati Children’s Hospital Medical Center, the research survey...


One of the most widely discussed symptoms of sickle cell anemia is a sickle cell crisis. This is an episode of pain caused when sickle-shaped red blood cells block the body’s blood vessels and prevent oxygen from flowing. One can read the biological explanations of such a crisis, but can’t really understand what it feels like without having the disease. So, it is only natural for people to wonder about it. I’m constantly asked what...


The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to CTX001, an investigational gene-editing cell therapy, for the treatment of severe sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT). The announcement was made by CRISPR Therapeutics and Vertex Pharmaceuticals, which are working together to develop CTX001. RMAT is given to expedite the development and review of new therapies whose main purpose is to treat medical conditions considered serious or...


Please consider donating to our non-profit such that we can continue building out support for the community.

May 2020
« Apr