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PMM2-CDG

Clinical trials for PMM2-CDG explained in plain language.

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Not yet finished but already full! 2 Completed 1 Terminated 1
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  • Could a diabetes drug help kids with rare PMM2-CDG?

    Disease control Terminated

    This study tested an oral drug called epalrestat, originally used for diabetic nerve pain, in children with PMM2-CDG, a rare genetic disorder that causes nerve problems and other symptoms. The trial aimed to see if the drug could safely improve nerve function and metabolic marker…

    Matched conditions: PMM2-CDG

    Phase: PHASE3 • Sponsor: Maggie's Pearl, LLC • Aim: Disease control

    Last updated Jun 27, 2026 07:52 UTC

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