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Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships

Crossref DOI link: https://doi.org/10.1186/s13023-015-0344-4

Published Online: 2015-10-07

Published Print: 2015-12

Update policy: https://doi.org/10.1007/springer_crossmark_policy

Authors

Al-Sannaa, Nouriya A.

Bay, Luisa

Barbouth, Deborah S.

Benhayoun, Youssef

Goizet, Cyril

Guelbert, Norberto

Jones, Simon A.

Kyosen, Sandra Obikawa

Martins, Ana Maria

Phornphutkul, Chanika

Reig, Celia

Pleat, Rebecca

Fallet, Shari

Ivanovska Holder, Iva