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

Promising PMM2-CDG drug trial halted early

Disease control Terminated

This study tested an oral drug called epalrestat in children aged 2 to 17 with PMM2-CDG, a rare genetic disorder that affects how the body makes certain proteins. The goal was to see if the drug could improve symptoms and lab markers compared to a placebo. The trial was terminate…

Matched conditions: PMM2-CDG

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

Last updated Apr 24, 2026 16:05 UTC

PMM2-CDG

Get alerted when new PMM2-CDG trials appear

Promising PMM2-CDG drug trial halted early