Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)

2020 ◽  
Vol 129 (2) ◽  
pp. S56-S57 ◽  
Author(s):  
Kevin M. Flanigan ◽  
Nicholas J.C. Smith ◽  
Maria Luz Couce ◽  
Kristen V. Truxal ◽  
Kim L. McBride ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Gene Therapy ◽  
Single Dose ◽  
Phase 1 ◽  
Type A ◽  
Syndrome Type ◽  
Mucopolysaccharidosis Type ◽  
Sanfilippo Syndrome ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

Scoring Evaluation of the Natural Course of Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A)

PEDIATRICS ◽  
2007 ◽  
Vol 120 (5) ◽  
pp. e1255-e1261 ◽  
Author(s):  
A. Meyer ◽  
K. Kossow ◽  
A. Gal ◽  
C. Muhlhausen ◽  
K. Ullrich ◽  
...  
Keyword(s):  
Type A ◽  
Natural Course ◽  
Syndrome Type ◽  
Mucopolysaccharidosis Type ◽  
Sanfilippo Syndrome ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

Natural history of siblings with mucopolysaccharidosis type IIIA, Sanfilippo syndrome type A

2017 ◽  
Vol 120 (1-2) ◽  
pp. S57-S58
Author(s):  
Alexandra Gossler ◽  
Anja Koehn ◽  
Ann Meyer ◽  
Kai Kossow ◽  
Kurt Ullrich ◽  
...  
Keyword(s):  
Natural History ◽  
Type A ◽  
Syndrome Type ◽  
Mucopolysaccharidosis Type ◽  
Sanfilippo Syndrome ◽  
Type Iiia ◽  
History Of ◽  
Mucopolysaccharidosis Type Iiia

Updated results of Transpher A: multicenter, single-dose, phase 1/2 clinical trial of ABO-102 for Sanfilippo syndrome type A (mucopolysaccharidosis IIIA)

2021 ◽  
Vol 132 ◽  
pp. S133-S134
Author(s):  
Kevin Flanigan ◽  
Nicholas Smith ◽  
Maria Couce ◽  
Maria Escolar ◽  
Kristen Truxal ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Single Dose ◽  
Phase 1 ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome

Updated results of Transpher B: multicenter, single-dose, phase 1/2 clinical trial of ABO-101 for Sanfilippo syndrome type B (mucopolysaccharidosis IIIB)

2021 ◽  
Vol 132 ◽  
pp. S139
Author(s):  
Maria de Castro ◽  
Kevin Flanigan ◽  
Bénédicte Héron ◽  
Nicole Muschol ◽  
Maria Couce ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Single Dose ◽  
Phase 1 ◽  
Syndrome Type ◽  
Type B ◽  
Sanfilippo Syndrome

Design and rationale of the LYS-SAF302 gene therapy study in mucopolysaccharidosis type IIIA (MPS IIIA) children

2019 ◽  
Vol 126 (2) ◽  
pp. S111-S112
Author(s):  
Sophie Olivier ◽  
Samantha Parker ◽  
Anupam Chakrapani ◽  
Ronald Crystal ◽  
Lisa Emrick ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mps Iiia ◽  
Mucopolysaccharidosis Type Iiia

Lentiviral mediated stem cell gene therapy corrects a mouse model of mucopolysaccharidosis type IIIA

2011 ◽  
Vol 102 (2) ◽  
pp. S8
Author(s):  
Brian Bigger ◽  
Alex Langford-Smith ◽  
Fiona Wilkinson ◽  
Kia Langford-Smith ◽  
Ana Sergijenko ◽  
...  
Keyword(s):  
Gene Therapy ◽  
Stem Cell ◽  
Mouse Model ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Cell Gene ◽  
Mucopolysaccharidosis Type Iiia ◽  
Stem Cell Gene ◽  
Stem Cell Gene Therapy
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