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Mucopolysaccharidosis II - 30 Studies Found

Status Study
Active, not recruiting Study Name: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy
Condition:
  • Mucopolysaccharidosis Type III A
  • Sanfilip
    Date: 2014-01-16
    Interventions: Genetic: SAF-301
Completed Study Name: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome
Condition:
  • Mucopolysaccharidosis Type III A
  • Sanfilip
    Date: 2011-11-10
    Interventions: Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod
Completed Study Name: Biomarker for Sanfilippo Disease
Condition:
  • Mucopolysaccharidosis Type 3 A
  • Mucopolysaccharidosis Type 3 B
  • Sanfilippo Syndrome
    Date: 2016-03-17
    Interventions: Biological: scAAV9.U1a.hSGSH Self-complementary adeno-associated virus serotype 9 carrying the human SGS
Enrolling by invitation Study Name: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093.
Condition:
  • Sanfilippo Syndrome
  • Mucopolysaccharidosis (MPS)

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