Mucopolysaccharidosis Type IIIA - 19 Studies Found
Completed |
: Intracerebral Gene Therapy for Sanfilippo Type A Syndrome :
: 2011-11-10 : Genetic: SAF-301 The treatment plan consists on a direct injection of the investigational medicinal prod |
Enrolling by invitation |
: An Extension Study to Determine Safety and Efficacy for Pediatric Patients With MPS Type IIIA Disease Who Participated in Study HGT-SAN-093. :
: 2015-01-21 : Drug: HGT-1410 HGT-1410 administered according to Patient Group assignment. |
Recruiting |
: A Study of Mucopolysaccharidosis Type IIIB (MPS IIIB) :
|
Recruiting |
: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children :
|
Terminated |
: ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases :
|
Completed |
: HSCT for High Risk Inherited Inborn Errors :
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RECRUITING |
: Study of DNL126 in Pediatric Participants With Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) : Mucopolysaccharidosis Type IIIA : 2024-05-13 : intravenous repeating dose |