Growth charts for patients with Sanfilippo syndrome (Mucopolysaccharidosis type III)
Abstract Background Mucopolysaccharidosis (MPS) type III (Sanfilippo syndrome) comprises a group of rare, lysosomal storage diseases caused by the deficiency of one of four enzymes involved in the degradation of heparan sulfate. The clinical hallmark of the disease is severe neurological deteriorati...
Main Authors: | , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
BMC
2019-05-01
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Series: | Orphanet Journal of Rare Diseases |
Subjects: | |
Online Access: | http://link.springer.com/article/10.1186/s13023-019-1065-x |