Neither cytokine- nor coagulopathy-related abnormalities were observed. Expression for the fifth patient was 17.5% of normal control samples. In addition, the level of serum creatine kinase decreased from baseline. The six patients dosed in IGNITE DMD ranged in age from five to fourteen at baseline. Mission Our mission, which guides every aspect of our operations, is to cure Duchenne. Genes are prone to mutations, which can either be inherited or occur spontaneously. At 2E14 vg/kg, the level of expression ranged from 30% to 70% of wild-type dystrophin. Systemic administration of SGT-001 was generally well tolerated in both species. We believe this event was attributed to procedural errors. In October 2017, the first milestone was achieved under this agreement. We have the first right to enforce such licensed patents at our expense. We recorded the upfront license fee as a research and development expense in 2016. The Company has made no payments to Ultragenyx during the year ended December 31, 2020. The FDA has agreed to specified performance goals in the review of BLAs under the PDUFA. Additional guidances are expected to be finalized by the FDA. 8226; Breakthrough therapy designation. Accelerated evaluation may be granted by the CHMP in exceptional cases. Thereafter, the U.S. Supreme Court agreed to hear this case. Oral argument in the case took place on November 10, 2020. A ruling by the Court is expected sometime this year. In addition, our product candidates, if approved, may not achieve commercial success. In October 2020, the FDA lifted the clinical hold placed on IGNITE DMD. Designation as a breakthrough therapy is within the discretion of the FDA. We might not receive such designation for our other product candidates. We currently have no medical affairs team. A broad range of legislative measures also have been introduced at the federal level.