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Biomedicine & Pharmacotherapy, 141 , Article 111839

Compound Type: Modified Pentadecapeptide (BPC-157 analogue with Arginine modification)

Carnitine uptake defect due to a 5UTR mutation in a pedigree with false positives and false negatives on Newborn screening

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A potential treatment regimen might include: (1) a short course of immunomodulation (e.g., with a biologic drug) to suppress the active autoimmune response, followed by (2) transplantation of stem cell-derived beta cells, potentially gene-edited and engineered to evade the immune system, possibly combined with (3) Treg adjuvant therapy to maintain long-term tolerance [133]