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Mucopolysaccharidosis Type IIIA - 19 Studies Found
Estado | Estudiar |
Completed |
Nombre del estudio: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome Condición:
Fecha: 2011-11-10 Intervenciones: Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod |
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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Recruiting |
Nombre del estudio: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children Condición:
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Terminated |
Nombre del estudio: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases Condición:
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Completed |
Nombre del estudio: HSCT for High Risk Inherited Inborn Errors Condición:
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RECRUITING |
Nombre del estudio: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) Condición: Mucopolysaccharidosis Type IIIA Fecha: 2024-05-18 Intervenciones: intravenous repeating dose |