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Mucolipidosis Type IV
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucolipidosis Type IV trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucolipidosis Type IV trials you may qualify forThe primary objectives of the study are: to describe the characteristics of the current international MLIV population; to define the median age at which patient…
Safety and Efficacy of AAV9.hMCOLN1co for patients with Mucolipidosis Type IV(MLIV): A Single-Center, Interventional, Open-Label, Single-Arm Clinical Study. The…
The purpose of this study is to define the natural history of Mucolipidosis Type IV and identify potential clinical outcome measures.