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Patient families expressed the following: Long-term goal is independenceFunctional independence and personal autonomy are key outcomes with the greatest impact for improving quality of life Incremental therapiesFirst slow, then halt, disease progression, then work to reverse symptoms Sustainable therapiesLifelong diseases need lifelong treatments, therefore the therapies we identify must be available to patients for as many years as they need, and have a minimized financial burden II

For gut and liver protection, consider adding oral BPC-157 as a supplemental route

A glutathione reductase from Escherichia coli