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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
by
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
Published in
Orphanet journal of rare diseases
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Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: Analysis of eight sibling pairs
by
Pleat, Rebecca
,
Al-Sannaa, Nouriya
,
Barbouth, Deborah S.
,
Benhayoun, Youssef
,
Goizet, Cyril
,
Guelbert, Norberto
,
Jones, Simon A.
,
Kyosen, Sandra
,
Martins, Ana Maria
,
Phornphutkul, Chanika
,
Reig, Celia
,
Holder, Iva Ivanovska
,
Fallet, Shari
Published in
Molecular genetics and metabolism
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Molecular Genetics And Metabolism
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Activities Of Daily Living
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Child
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Child, Preschool
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Clinical Outcomes
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Early Diagnosis
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Iduronidase - Therapeutic Use
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