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Mucopolysaccharidosis Type I - 128 Studies Found
Recruiting |
: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) :
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Active, not recruiting |
: A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously :
: 2015-10-21 : Drug: SBC-103 |
Not yet recruiting |
: Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III :
: Other: This is an observational study This is an observational study |
Recruiting |
: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children :
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Active, not recruiting |
: Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders :
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Terminated |
: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases :
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