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

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 Sanfilippo syndrome type A

2013 ◽  
Vol 37 (3) ◽  
pp. 431-437 ◽  
Author(s):  
Dakota Buhrman ◽  
Kavita Thakkar ◽  
Michele Poe ◽  
Maria L. Escolar
Keyword(s):  
Natural History ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
History Of

The natural history of Sanfilippo syndrome type A

2013 ◽  
Vol 108 (2) ◽  
pp. S90
Author(s):  
Kavita Thakkar ◽  
Dakota Buhrman ◽  
Michele Poe ◽  
Maria L. Escolar
Keyword(s):  
Natural History ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
History Of

Design and rational of the natural history study in preparation for the pivotal clinical trial for the treatment of Sanfilippo syndrome type A

2017 ◽  
Vol 120 (1-2) ◽  
pp. S25-S26
Author(s):  
Soraya Bekkali ◽  
Samantha Parker ◽  
Anupam Chakrapani ◽  
Roberto Giugliani ◽  
Benedicte Heron ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Natural History ◽  
Type A ◽  
Syndrome Type ◽  
Sanfilippo Syndrome ◽  
Natural History Study ◽  
Pivotal Clinical Trial

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 Sanfilippo syndrome type B in young patients: Ongoing results from two large, prospective studies

2021 ◽  
Vol 132 (2) ◽  
pp. S44
Author(s):  
Roberto Giugliani ◽  
Ilyas Okur ◽  
Fatih Ezgu ◽  
Nicole Muschol ◽  
Paul Harmatz ◽  
...  
Keyword(s):  
Natural History ◽  
Prospective Studies ◽  
Young Patients ◽  
Syndrome Type ◽  
Type B ◽  
Sanfilippo Syndrome ◽  
History Of
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