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Mucopolysaccharidosis Type I - 128 Studies Found
Estado | Estudiar |
Completed |
Nombre del estudio: Natural History Study of Patients With Mucopolysaccharidosis Type IIIB (MPS IIIB, Sanfilippo Syndrome Type B) Condición: Sanfilippo Syndrome Type B Fecha: 2012-01-10 |
Completed |
Nombre del estudio: Neurobehavioral Phenotypes in MPS III Condición:
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Recruiting |
Nombre del estudio: A Treatment Study of Mucopolysaccharidosis Type IIIB Condición:
Fecha: 2016-02-27 Intervenciones: Drug: BMN 250 Chimeric fusion of recombinant human alpha-N-acetylglucosaminidase and truncated human ins |
Completed |
Nombre del estudio: Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients Condición: Sanfilippo Syndrome Type A (MPS IIIA) Fecha: 2010-06-24 Intervenciones:
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Recruiting |
Nombre del estudio: Biomarker for Sanfilippo Disease Condición:
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Completed |
Nombre del estudio: Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease Condición: Sanfilippo Syndrome Fecha: 2014-02-10 Intervenciones: Drug: Recombinant human heparan N-sulfatase [rhHNS] Recombinant human heparan N-sulfatase [rhHNS] |
Completed |
Nombre del estudio: A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB Condición: MPS IIIB (Sanfilippo Syndrome) Fecha: 2014-11-03 |
Completed |
Nombre del estudio: Natural History Studies of Mucopolysaccharidosis III Condición:
Fecha: 2013-11-26 Intervenciones:
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Recruiting |
Nombre del estudio: Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH for Mucopolysaccharidosis (MPS) IIIA Condición: Mucopolysaccharidosis Type 3 A Sanfilippo Syndrome Fecha: 2016-03-17 Intervenciones: Biological: scAAV9.U1a.hSGSH Self-complementary adeno-associated virus serotype 9 carrying the human SGS |
Enrolling by invitation |
Nombre del estudio: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. Condición:
Fecha: 2015-01-21 Intervenciones: Drug: HGT-1410 HGT-1410 administered according to Patient Group assignment. |