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Mucopolysaccharidosis Type III - 55 Studies Found
Estado | Estudiar |
Completed |
Nombre del estudio: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB Condición: MPS IIIB (Sanfilippo Syndrome) Fecha: 2014-11-03 |
Completed |
Nombre del estudio: Natural History Studies of Mucopolysaccharidosis III Condición:
Fecha: 2013-11-26 Intervenciones:
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Recruiting |
Nombre del estudio: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA Condición: Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome Fecha: 2016-03-17 Intervenciones: Biological: scAAV9.U1a.hSGSH Self-complementary adeno-associated virus serotype 9 carrying the human SGS |
Enrolling by invitation |
Nombre del estudio: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. Condición:
Fecha: 2015-01-21 Intervenciones: Drug: HGT-1410 HGT-1410 administered according to Patient Group assignment. |
Recruiting |
Nombre del estudio: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) Condición:
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Active, not recruiting |
Nombre del estudio: 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 Condición:
Fecha: 2015-10-21 Intervenciones: Drug: SBC-103 |
Not yet recruiting |
Nombre del estudio: Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III Condición:
Intervenciones: Other: This is an observational study This is an observational study |
Recruiting |
Nombre del estudio: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children Condición:
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