An update from John Otsuki, SCDAA government relations manager As I am sure you’re aware, the United States recently completed a midterm election. All members of the House of Representatives were up for election or re-election, as they are every 2 years, as were 34 Senators (roughly 1/3 of the total). Before the election, the Democrats controlled both the Senate and the House, both by.....

This weekend’s Saturday Night Live skit about the recent historic approvals of potentially curative gene therapies for sickle cell disease is distasteful at best and harmful at worst. Earlier this month, the Food and Drug Administration approved groundbreaking new treatments that could change the lives of thousands. SNL chose to cast a spotlight on this news with a tone-deaf skit depicting a workplace Yankee Swap.....

Maryland Governor Wes Moore has signed a proclamation to make June 19, 2023, Sickle Cell Awareness Day! This recognition goes a........

Dublin, Ireland, 19 June 2023: Afimmune, a clinical stage biopharmaceutical company developing novel rare disease therapeutics, today announced it has.....

Two gene therapies were recently approved by the Food and Drug Administration (FDA) to treat sickle cell disease: Casegevy from CRISPR/Vertex and Lyfgenia from bluebird bio. You probably have questions about these new treatment options. Read more below. Is gene therapy a cure for sickle cell disease? Gene therapy is a potentially curative therapy. This means that it could act as a cure, but it.....

We are very excited to share that today, Dec. 8, the Food and Drug Administration approved two gene therapies to treat sickle cell disease! These potentially curative therapies are the first treatments of their kind available to individuals with SCD. We are heartened by this approval and are proud to support our community during this milestone moment. SCDAA will be releasing a full statement and.....

Today, Dec. 8, the Food and Drug Administration approved exa-cel gene therapy for sickle cell disease, making it the first treatment of its kind available to individuals with SCD in the United States. The approval of this potentially curative therapy marks a major advance in the treatment of sickle cell disease; however, there are valid concerns about accessibility and the potential for adverse effects. SCDAA.....

Exa-cel gene therapy was recently approved by the Food and Drug Administration to treat sickle cell disease. You probably have questions about this new treatment option. Read more below. Is this a cure for sickle cell disease? Exa-cel is a potentially curative therapy. This means that it could act as a cure, but it is too new to say for sure. Exa-cel causes a big.....

On Dec. 8, 2023, the Food and Drug Administration (FDA) approved exagamglogene autotemcel (exa-cel) gene therapy for sickle cell disease (SCD), making it the first treatment of its kind available to individuals with SCD in the United States. The approval of this potentially curative therapy marks a major advance in the treatment of sickle cell disease; however, there are valid concerns about accessibility and the.....