Two breakthrough gene therapies can now be used to treat and possibly cure sickle cell anemia, the genetic blood disorder that afflicts 100,000 mostly Black Americans and 20 million people worldwide.
But the announcement from the Food and Drug Administration of approval of the treatments the first use of medicines to address an inherited disease drew cheers and caution flags from those in the field.
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- Continued strong commercial launch for ZYNTEGLO® and SKYSONA®; 22 patient starts across both programs to date –
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Oral presentation details efficacy, safety and health-related quality of life data in adult and pediatric patients with sickle cell disease treated with lovo-cel in HGB-206 Group C and HGB-210 .