Mucopolysaccharidosis II - 50 Studies Found
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 |
Completed |
: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease : Sanfilippo Syndrome : 2014-02-10 : Drug: Recombinant human heparan N-sulfatase [rhHNS] Recombinant human heparan N-sulfatase [rhHNS] |
Completed |
: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB : MPS IIIB (Sanfilippo Syndrome) : 2014-11-03 |
Active, not recruiting |
: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy :
|
Completed |
: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome :
|
Recruiting |
: Biomarker for Sanfilippo Disease :
|
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 Treatment Study of Mucopolysaccharidosis Type IIIB :
: 2016-02-27 : Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins |
Completed |
: Neurobehavioral Phenotypes in MPS III :
|