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Mucopolysaccharidosis IIIA
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IIIA trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Mucopolysaccharidosis IIIA trials may be worth asking aboutNorth America
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The main objective of this study is to evaluate the efficacy and safety of UX111 for the treatment of MPS IIIA.
The purpose of this study is to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of recombinant human heparan N-sulfatase (rhH…
The main objective of this study is to evaluate the safety/tolerability and efficacy of UX111 (previously known as ABO-102) in participants with Mucopolysacchar…
This is a multicenter, open-label, Phase 1/2 study to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and clinical efficacy of DNL…