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SMARD1
Clinical trial pipeline · Data from ClinicalTrials.gov
See which SMARD1 trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which SMARD1 trials may be worth asking aboutNorth America
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Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
This is an 'N of 1', open-label, single center study to evaluate the safety of therapy with VCA-894A, an ASO designed to rescue and restore the activity of IGHM…
Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.