Mucopolysaccharidosis I - 56 Studies Found

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
  • Morquio Disease
  • Mucopolysaccharidosis IV<
    : 2012-12-13
Terminated : Morquio's Syndrome: a Case Study
: Morquio's Disease
: 2008-01-25
: Other: physiotherapeutic treatment At first, the patient was submitted to a conventional physiotherapeut
Recruiting : Pregnancy With Morquio Syndrome - What Are Patients' Perspectives and Has ERT Changed Them?
:
  • Morquio Disease
  • MPS - Mucopolysaccharidosis

: 2017-05-10
: Other: Interview In-person or telephone in-depth interview
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