An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA-Reference-Cited by-同舟云学术

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

Published:2022-02 Issue:2 Volume:135 Page:133-142
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

Wijburg Frits A.,Aiach Karen,Chakrapani Anupam,Eisengart Julie B.,Giugliani Roberto,Héron Bénédicte,Muschol Nicole,O'Neill Cara,Olivier Sophie,Parker Samantha

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

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4. Scoring evaluation of the natural course of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A);Meyer;Pediatrics,2007

5. Challenging symptoms in children with rare life-limiting conditions: findings from a prospective diary and interview study with families;Malcolm;Acta Paediatr.,2012

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