scholarly journals An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA

2021 ◽  
Author(s):  
Frits A. Wijburg ◽  
Karen Aiach ◽  
Anupam Chakrapani ◽  
Julie B. Eisengart ◽  
Roberto Giugliani ◽  
...  
Keyword(s):  
Natural History ◽  
Mucopolysaccharidosis Type ◽  
Natural History Study ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

scholarly journals A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA

2016 ◽  
Vol 170 ◽  
pp. 278-287.e4 ◽  
Author(s):  
Elsa G. Shapiro ◽  
Igor Nestrasil ◽  
Kathleen A. Delaney ◽  
Kyle Rudser ◽  
Victor Kovac ◽  
...  
Keyword(s):  
Natural History ◽  
Mucopolysaccharidosis Type ◽  
Natural History Study ◽  
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

scholarly journals Cardiac disease as the presenting feature of mucopolysaccharidosis type IIIA: A case report

2014 ◽  
Vol 1 ◽  
pp. 422-424 ◽  
Author(s):  
Erlane Marques Ribeiro ◽  
Ana Carolina Brusius-Facchin ◽  
Sandra Leistner-Segal ◽  
Carlos Antônio Bruno da Silva ◽  
Ida Vanessa Schwartz
Keyword(s):  
Case Report ◽  
Cardiac Disease ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

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

scholarly journals Pentosan Polysulfate Treatment of Mucopolysaccharidosis Type IIIA Mice

2018 ◽  
pp. 37-52 ◽  
Author(s):  
Ningning Guo ◽  
Victor DeAngelis ◽  
Changzhi Zhu ◽  
Edward H. Schuchman ◽  
Calogera M. Simonaro
Keyword(s):  
Mucopolysaccharidosis Type ◽  
Pentosan Polysulfate ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia
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