A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

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 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 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 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)