PHOSPHOMANNOMUTASE 2 DEFICIENCY
Clinical trials for PHOSPHOMANNOMUTASE 2 DEFICIENCY explained in plain language.
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Hope for rare disease: new drug shows promise in Long-Term study
Disease control ENROLLING_BY_INVITATIONThis study is for people with PMM2-CDG, a rare genetic disorder, who have already taken the experimental drug GLM101 in a previous trial. The goal is to see if GLM101 is safe and effective over a longer period. Participants will receive weekly infusions of GLM101 at the same dose…
Matched conditions: PHOSPHOMANNOMUTASE 2 DEFICIENCY
Phase: PHASE2 • Sponsor: Glycomine, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:10 UTC
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Experimental drug GLM101 targets rare PMM2-CDG in pivotal trial
Disease control OngoingThis study tests a drug called GLM101 for people with PMM2-CDG, a rare inherited disease that affects movement and coordination. About 50 children and adults will receive weekly infusions of either GLM101 or a placebo for 24 weeks, followed by an open-label phase where everyone g…
Matched conditions: PHOSPHOMANNOMUTASE 2 DEFICIENCY
Phase: PHASE2, PHASE3 • Sponsor: Glycomine, Inc. • Aim: Disease control
Last updated Jun 27, 2026 09:08 UTC
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Researchers track rare disease PMM2-CDG to unlock clues for future treatments
Knowledge-focused OngoingThis study is gathering medical information from 120 people with PMM2-CDG, a rare genetic disorder. Researchers will track growth, organ function, and development over time. The goal is to better understand the disease and help design future treatments. No new drugs are being tes…
Matched conditions: PHOSPHOMANNOMUTASE 2 DEFICIENCY
Sponsor: Glycomine, Inc. • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:57 UTC