Phase I/II Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
study id #: NCT02716246
condition: MPS IIIA, Sanfilippo Syndrome, Sanfilippo A, Mucopolysaccharidosis III
status: Recruiting
purpose:Open-label, dose-escalation clinical trial of scAAV9.U1a.hSGSH injected intravenously through a peripheral limb vein
intervention: ABO-102
results: https://clinicaltrials.gov/ct2/show/results/NCT02716246
last updated: February 26, 2022
-
Awake Airway Endoscopy in Mucopolysaccharidosis: A Case ReportMucopolysaccharidosis (MPS) are a group ...
-
Denali Therapeutics Announces Continued Progress in DNL310 (ETV:IDS) Program for MPS II (Hunter Syndrome) Supporting...Denali Therapeutics Inc., a biopharmaceu...
-
Epidemiology and Genetics of Mucopolysaccharidosis Type VI in RussiaMucopolysaccharidosis VI (MPS VI) is an ...
-
Molecular Analysis and Novel Variation Identification of Chinese Pedigrees With Mucopolysaccharidosis Using Targeted...Mucopolysaccharidosis (MPS) refers to a ...
-
Emotional Responses to Diagnosis of MPS or MLIf you or someone you know have been dia...
-
What Is MPS IX or Natowicz Disease?MPS IX, known as Natowicz disease, is on...
-
Inhibition of Iduronic Acid Biosynthesis by Ebselen Reduces Glycosaminoglycan Accumulation in Mucopolysaccharidosis ...Mucopolysaccharidosis type I (MPS-I) is ...