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Acta Pharm Sin B (2016) 6(5):41325

doi: 10.1097/bor.0000000000000373 35

There is a regular feedback of errors from the DMC to the study sites

Afterwards, a second incubation was made under the previous conditions

Current therapies include ERT to compensate for the missing or defective enzyme, and substrate reduction therapy to reduce the accumulation of toxic substances by limiting their production