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Consensus statement on enzyme replacement therapy for mucopolysaccharidosis IVA in Central and South-Eastern European countries.

Martin MagnerZsuzsanna AlmássyZoran GucevBeata Kieć-WilkVasilica PlaiasuAnna Tylki-SzymańskaDimitrios ZafeiriouIoannis ZaganasChristina Lampe
Published in: Orphanet journal of rare diseases (2022)
European guidelines and evidence-based recommendations for Morquio A patients should be considered in the real life of Central and South-Eastern European countries and adapted to unique clinical practice approaches and criteria for patients' access to treatment and reimbursement in the region.
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