Vertex Pharmaceuticals and CRISPR Therapeutics are planning a November launch for a biologics license application (BLA) for their gene-editing therapy exagamglogene autotemcel — known as exa-cel — seeking its approval for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia. The U.S. Food and Drug Administration (FDA)…
News
Long-term treatment with Ferriprox (deferiprone) safely and effectively reduced iron load in children and adults with blood transfusion-induced iron overload due to sickle cell disease (SCD) or other anemias. That is according to three years of data from a one-year Phase 4 clinical trial, called FIRST (NCT02041299), and its…
Children with sickle cell disease (SCD) are at a high risk of developing severe symptoms after infection with the SARS-CoV-2 Omicron variant, one of the most recent variants of the virus that causes COVID-19, according to a new French study. In fact, more than 85% of children infected with…
Global Blood Therapeutics (GBT) supports the recently introduced Sickle Cell Disease Treatment Centers Act of 2022 (HR 8855/S 4866), which calls for $535 million in annual funding for sickle cell disease (SCD) treatment, research, and education. Introduced on Sept. 15, the legislation would create a national network of…
A new cost-free program, called Sickle Cell Warrior program, designed to promote equitable care and provide information on clinical trials and financial assistance to sickle cell disease (SCD) patients has been launched by The National Marrow donor program (NMDP)/Be The Match. As part of this month’s SCD Awareness…
The 11th Annual Sickle Cell Disease Therapeutics Conference, to be held virtually on Sept. 14 — at the heart of this National SCD Awareness Month — will focus on the latest advances and future trends for treating patients with sickle cell disease (SCD). The…
To help bring awareness this month to sickle cell disease (SCD) and childhood cancers, the supplemental insurance provider Aflac recently engaged young patients with those disorders at Nicklaus Children’s Hospital in south Florida through its My Special Aflac Duck social robot program. September is National Sickle Cell Awareness…
Forma Therapeutics, which is currently developing an experimental treatment for sickle cell disease (SCD) called etavopivat, is being acquired by Novo Nordisk, the two companies announced. “Novo Nordisk has worked for more than 40 years to develop and deliver transformative medicines to patients around the world with…
The National Institutes of Health (NIH) has awarded a five-year, $7.7-million grant to support a new research project that seeks to lower barriers to healthcare for people with sickle cell disease (SCD). The project, titled “Recruitment and Engagement in Care to Impact Practice Enhancement (RECIPE) for Sickle…
For most of her life, 38-year-old DeeAnn Edwards Mann has been quiet about having sickle cell disease (SCD). She has tried to remain anonymous on sickle cell-related forums and social media conversations, and doesn’t talk about it much with her friends. In some cases, she can even hide her…
Recent Posts
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