Mucopolysaccharidosis I - 56 Studies Found Completed : Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II) : Mucopolysaccharidosis (MPS) Hunter Syndrome : 2013-03-12 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 : Biological: rhHNS-10 mg Once p 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 : Biological: Recombinant human heparan N-sulfatase 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 : Mucopolysaccharidosis III, Type B (MPS IIIB) Sanfilippo B : 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 : Mucopolysaccharidosis Type III A Sanfilip : 2014-01-16 : Genetic: SAF-301 Completed : Intracerebral Gene Therapy for Sanfilippo Type A Syndrome : Mucopolysaccharidosis Type III A Sanfilip : 2011-11-10 : Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod <<< Previous | Next >>>
