Mucopolysaccharidosis Type I - 128 Studies Found
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] |
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 :
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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. |