Lipid profile in adult patients with Fabry disease - Ten-year follow up

por Stepien, Karolina M., Hendriksz, Chris J.
Publicado 2017

Niemann–Pick type C disease – the tip of the iceberg? A review of neuropsychiatric presentation, diagnosis and treatment

por Evans, William R. H., Hendriksz, Chris J.
Publicado 2017

Mortality in patients with Sanfilippo syndrome

por Lavery, Christine, Hendriksz, Chris J., Jones, Simon A.
Publicado 2017

The factors affecting lipid profile in adult patients with Mucopolysaccharidosis

por Stepien, Karolina M., Stewart, Fiona J., Hendriksz, Chris J.
Publicado 2017

Recommendations for patient screening in ultra-rare inherited metabolic diseases: what have we learned from Niemann-Pick disease type C?

por Sobrido, María-Jesús, Bauer, Peter, de Koning, Tom, Klopstock, Thomas, Nadjar, Yann, Patterson, Marc C, Synofzik, Matthis, Hendriksz, Chris J
Publicado 2019

A systematic review of the prevalence of Morquio A syndrome: challenges for study reporting in rare diseases

por Leadley, Regina M, Lang, Shona, Misso, Kate, Bekkering, Trudy, Ross, Janine, Akiyama, Takeyuki, Fietz, Michael, Giugliani, Roberto, Hendriksz, Chris J, Hock, Ngu Lock, McGill, Jim, Olaye, Andrew, Jain, Mohit, Kleijnen, Jos
Publicado 2014