Mucopolysaccharidosis III - 16 Studies Found
| Active, not recruiting |
: Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) : Sanfilippo Syndrome : 2011-02-16 :
|
| Completed |
: A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA) : Sanfilippo Syndrome Type A : 2010-01-11 : Other: assessment Physical, developmental, neurological, behavioral, and neurocognitive assessments |
| Completed |
: Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) : Sanfilippo Syndrome Type B : 2012-01-10 |
| Completed |
: Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients : Sanfilippo Syndrome Type A (MPS IIIA) : 2010-06-24 :
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| 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 :
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