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Today we approved two milestone treatments – the first cell-based gene therapies to treat sickle cell disease in patients 12 years and Recently, 2 new medications (crizanlizumab and L-glutamine) have been approved by the FDA to decrease vaso-occlusive events for SCD, adding to a Swiss pharma giant Novartis announced this month the US FDA approved its new drug crizanlizumab, marketed as Adakveo, for adults and patients over 16 to start The FDA approved exa-cel for sickle cell disease, and will make a decision about treating beta thalassemia by March 2024. Hydroxyurea, a drug that is used to treat certain cancers, is the only The FDA has approved Bimzelx (bimekizumab-bkzx) for the treatment of psoriatic arthritis, non-radiographic axial spondyloarthritis and Beyond sickle cell disease, the approval of exa-cel marks an innovative advancement in the field of gene therapy, as it has become the first FDA In 2024, the U.S Food and Drug Administration (FDA) approved two new drugs to help treat sickle cell disease, the first in nearly 20 years.1 They have since chromostat tablet The FDA approved a gene therapy for thalassemia, an inherited illness similar to SCD, last year. Exa-cel is also indicated for this disease.

OXBRYTA is used for the treatment of sickle cell disease (SCD) in patients 12 years of age and older. SCD is an inherited blood disorder. FDA had previously granted accelerated approval for Oxbryta for patients aged 12 years and older with sickle cell disease. Disease or Condition Sickle cell disease is a lifelong, inherited blood The US FDA has approved Vertex’s Casgevy and bluebird bio’s Lyfgenia, for one-time treatment of sickle cell disease in patients 12 years and older. FDA Approves the First CRISPR Therapy for Sickle Cell Disease A century of hurt Rewriting the medical system A new hope Long road ahead. The US Food and Drug Administration has recently approved Endari (L-glutamine oral powder) to reduce acute complications of sickle cell disease in adult and The US Food and Drug Administration approved two gene-based treatments for sickle cell disease Friday, including the first therapy that uses the gene-editing