Mucopolysaccharidosis Type III - 55 Studies Found
Completed |
: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB : MPS IIIB (Sanfilippo Syndrome) : 2014-11-03 |
Completed |
: Natural History Studies of Mucopolysaccharidosis III :
: 2013-11-26 :
|
Recruiting |
: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA : Mucopolysaccharidosis Type 3 A 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. :
: 2015-01-21 : Drug: HGT-1410 HGT-1410 administered according to Patient Group assignment. |
Recruiting |
: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) :
|
Active, not recruiting |
: A Open Label Study in Previously Studied, SBC-103 Treatment Naïve MPS IIIB Subjects to Investigate the Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously :
: 2015-10-21 : Drug: SBC-103 |
Not yet recruiting |
: Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III :
: Other: This is an observational study This is an observational study |
Recruiting |
: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children :
|