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The accelerated approval was granted based on initial overall response rate (ORR) of 57% in 164 treatment-nave patients and duration of response (DOR)40% of responders had a duration of response greater than 12 months, according to the FDAs full approval announcement

This could be explained by frataxin reduction in different tissues being associated with distinct transcriptomic profiles, as described recently (Chandran et al., 2017)

3E and 6F)

Kreissl, A., Jorda, A., Truschner, K., Skacel, G

BAC Water is commonly used because: It is sterile It contains a preservative It allows for multiple withdrawals from the same vial It provides convenient storage and handling What Does Reconstitution Mean