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Achondroplasia
Disease
Literature
Materials
VOXZOGO
Literature
Materials
CLN2
Disease
Literature
Materials
BRINEURA
Literature
Materials
Haemophilia
Disease
Literature
Materials
ROCTAVIAN
Literature
Materials
MPS
Disease
MPS IV
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Materials
MPS VI
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NAGLAZYME
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VIMIZIM
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KUV/LT/013
Journal of Inherited Metabolic Disease (Vol. 30) Reprint
Burton B,. et al (2007) The response of patients with phenylketonuria and elevated serum phenylalanine to treatment with oral sapropterin dihydrochloride (6r-tetrahydrobiopterin): a phase II, multicentre, open label, screening study.
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EU-ACH-00163
Keep Up to Date with Evolving Management A5 Flyer
Promo Flyer Physicians
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EU/KUV/0232
Kuvan Tablet & Sachet Dosing Calculator - Germany
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VIM/LT/016
Melton (2017) Antibodies Elosulfase Alfa and Efficacy
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EU/MPS/1214
MPS Diagnostic Tool Folder
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CON/BIO/044
MPS Reference Website Lanyards (blue)
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BIO/EU/13/012
MPS VI (Polish)
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EU-PAL-00354
Palynziq Sales Aid Eucan
Sales aid for TMs to use to discuss with customers.
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PAL006
Patient Package Leaflet Spanish
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PKU/LT/005
Phenylketonuria in the Adult Patient
Ceberio L et al Expert Opinion on Orphan Drugs 2019:7 265-276
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PKU/BM/EN/ OCT 19 (Quarantine)
PKU Sampling Kit using Dried Blood Spot (DBS) Card
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EU-ACH-00367
Reaching A Consensus A5 Booklet
Consensus Publication Flyer for Physicians
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