Brain MRI reveals several abnormalities in patients with cerebral folate deficiency, including delayed myelin development, cerebellar atrophy, cortical laminar necrosis, bilateral intrabasal ganglia calcifications, frontal lobe and cerebellar atrophy
41 All studies used an intention-to-treat (ITT) analysis except for two in which this was unclear 42,43 and two that did not use an ITT analysis
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The human PK data gap As of 2026, no completed Phase 1 pharmacokinetic, Phase 2 efficacy, or Phase 3 confirmation trials of BPC-157 have been published in the peer-reviewed clinical trial literature
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