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Mucopolysaccharidosis II - 30 Studies Found

Estado Estudiar
Active, not recruiting Nombre del estudio: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Condición:
  • Mucopolysaccharidosis Type III A
  • Sanfilip
    Fecha: 2014-01-16
    Intervenciones: Genetic: SAF-301
Completed Nombre del estudio: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Condición:
  • Mucopolysaccharidosis Type III A
  • Sanfilip
    Fecha: 2011-11-10
    Intervenciones: Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod
Completed Nombre del estudio: Biomarker for Sanfilippo Disease
Condición:
  • Mucopolysaccharidosis Type 3 A
  • Mucopolysaccharidosis Type 3 B
  • 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:
  • Sanfilippo Syndrome
  • Mucopolysaccharidosis (MPS)

Fecha: 2015-01-21
Intervenciones: Drug: HGT-1410 HGT-1410 administered according to Patient Group assignment.
Completed Nombre del estudio: Evaluation of Blood Brain Barrier Integrity and Structural Abnormalities in MPS IIIB Patients Using Multimodal Magnetic Resonance Imaging
Condición: MPS IIIB (Sanfilippo B Syndrome)
Fecha: 2014-03-12
Recruiting Nombre del estudio: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)
Condición:
  • Mucopolysaccharidosis Type IIIB
  • Mucopolysaccharidosis Type 3 B