Mucopolysaccharidosis Type III - 55 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 :
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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 |
Active, not recruiting |
: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy :
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Completed |
: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome :
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Completed |
: Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) : Sanfilippo Syndrome Type B : 2012-01-10 |
Completed |
: Neurobehavioral Phenotypes in MPS III :
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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 |
: 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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Recruiting |
: Biomarker for Sanfilippo Disease :
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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] |