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Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group

  • Published:2020-06 Issue: Volume:23 Page:100578
  • ISSN:2214-4269
  • Container-title:Molecular Genetics and Metabolism Reports
  • language:en
  • Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Köhn Anja F.,Grigull Lorenz,du Moulin Marcel,Kabisch Sarah,Ammer Luise,Rudolph Cornelia,Muschol Nicole M.

Funder

Amicus

BioMarin

Sanofi Genzyme

Shire

Actelion

Chiesi Farmaceutici S.p.A.

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference30 articles.

1. Cumulative incidence rates of the mucopolysaccharidoses in Germany;Baehner;J. Inherit. Metab. Dis.,2005

2. A prospective natural history study of Mucopolysaccharidosis type IIIA;Shapiro;J. Pediatr.,2016

3. Scoring Evaluation of the Natural Course of Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A), Pediatrics;Meyer,2007

4. Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations;Valstar;Ann. Neurol.,2010

5. Mucopolysaccharidosis type III (Sanfilippo disease) in Sweden: clinical presentation of 22 children diagnosed during a 30-year period;Malm;Acta Paediatr. Int. J. Paediatr.,2010
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