Lipid profile in adult patients with Fabry disease - Ten-year follow up by Karolina M. Stepien, Chris J. Hendriksz

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Mortality in patients with Sanfilippo syndrome by Christine Lavery, Chris J. Hendriksz, Simon A. Jones

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The factors affecting lipid profile in adult patients with Mucopolysaccharidosis by Karolina M. Stepien, Fiona J. Stewart, Chris J. Hendriksz

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Recommendations for patient screening in ultra-rare inherited metabolic diseases: what have we learned from Niemann-Pick disease type C? by María-Jesús Sobrido, Peter Bauer, Tom de Koning, Thomas Klopstock, Yann Nadjar, Marc C Patterson, Matthis Synofzik, Chris J Hendriksz

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