Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH

Sanfilippo Syndrome

The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3) A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome) Natural History Observational Study of MPS IIIa in SMC A Long-term Follow-up Study of Patients With MPS IIIB Treated With ABO-101 A Prospective Natural History Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) Gene Transfer Study of ABO-102 in Patients With Middle and Advanced Phases of MPS IIIA Disease Study of AAVrh10-h.SGSH Gene Therapy in Patients With Mucopolysaccharidosis Type IIIA (MPS IIIA) Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) 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 Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH Natural History Study of Patients With MPS IIIA Parental Coping With Challenging Behavior in Mucopolysaccharidosis Type I-III An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients Gene Transfer Clinical Trial for Mucopolysaccharidosis (MPS) IIIB Natural History Studies of Mucopolysaccharidosis III Gene Therapy With Modified Autologous Hematopoietic Stem Cells for Patients With Mucopolysaccharidosis Type IIIA Long-term Follow-up of Sanfilippo Type A Patients Treated by Intracerebral SAF-301 Gene Therapy Intracerebral Gene Therapy for Sanfilippo Type A Syndrome Intracerebral Gene Therapy in Children With Sanfilippo Type B Syndrome A Long-term Follow-up Study of Patients With MPS IIIA Treated With ABO-102 Extension of Study HGT-SAN-055 Evaluating Administration of rhHNS in Patients With Sanfilippo Syndrome Type A (MPS IIIA) A Treatment Study of Mucopolysaccharidosis Type IIIB A Treatment Extension Study of Mucopolysaccharidosis Type IIIB Open-label Study of Anakinra in MPS III Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB)