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Mucopolysaccharidosis Type IIIA - 19 Studies Found
Estado | Estudiar |
Completed |
Nombre del estudio: A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA) Condición: Sanfilippo Syndrome Type A Fecha: 2010-01-11 Intervenciones: Other: assessment Physical, developmental, neurological, behavioral, and neurocognitive assessments |
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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Completed |
Nombre del estudio: Natural History Studies of Mucopolysaccharidosis III Condición:
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Completed |
Nombre del estudio: Neurobehavioral Phenotypes in MPS III Condición:
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Recruiting |
Nombre del estudio: Natural History Study of Patients With MPS IIIA Condición: Mucopolysaccharidosis IIIA Fecha: 2016-04-15 |
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] |
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 |
Active, not recruiting |
Nombre del estudio: Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) Condición: Sanfilippo Syndrome Fecha: 2011-02-16 Intervenciones:
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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 |
Active, not recruiting |
Nombre del estudio: Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy Condición:
Fecha: 2014-01-16 Intervenciones: Genetic: SAF-301 |