Mucopolysaccharidosis II - 29 Studies Found

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

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