Finding studies
Finding studies
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Lead
Glycomine, Inc.
This is a phase 2 open-label clinical study of GLM101 in patients with PMM2-CDG who have previously participated in a study of GLM101. This study is designed to monitor long-term safety and treatment effect of GLM101 and provide continued access to study treatment. Participants will receive 30 mg/kg. Dose levels may be adjusted to lower doses or higher doses based on available data that demonstrates a change to be safe and favorable. Among other assessments, participants will be asked to complete questionnaires to evaluate changes in ataxia and quality of life.
Age
2–any
Sex
ALL
Healthy volunteers
Not accepted
