July 7, 2023 – SCDAA’s Medical and Research Advisory Committee (MARAC) notes that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended on May 26, 2023, that the conditional marketing authorization for crizanlizumab (Adakveo) be revoked. The EMA stated that this revocation was triggered by the findings of the Novartis STAND multinational clinical trial showing no significant benefit of crizanlizumab.....

The Sickle Cell Disease Association of America, the leading patient organization for people with sickle cell disease, announced a pilot program with MedicAlert Foundation to enhance the safety and well-being of people living with sickle cell disease. More than 100,000 Americans live with sickle cell disease, a genetic blood disorder that affects red blood cells. Acute pain episodes known as sickle cell crises are one.....

Dublin, Ireland, 19 June 2023: Afimmune, a clinical stage biopharmaceutical company developing novel rare disease therapeutics, today announced it has been invited to join the Sickle Cell Disease Association of America’s (SCDAA) Collaboration of Advocates for Research, Education and Science (C.A.R.E.S.) Consortium. The mission of this initiative is to raise awareness about the importance of clinical trials and why it can help sickle cell patients.....

CMO Speaks is a blog featuring the voices of SCDAA’s clinical leadership team. Part 1 of our Gene Therapy series was written by Dr. Lewis Hsu and Dr. Sri Lakshmi Jamalapur. How can you cure an inherited disease like sickle cell disease? One way would be to develop some extremely effective medications that would alleviate all the symptoms. Another would be to transplant stem cells.....

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