Long-term clinical evaluation of patients with alpha-mannosidosis – A multicenter study
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Published:2024-04
Issue:
Volume:68
Page:104927
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ISSN:1769-7212
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Container-title:European Journal of Medical Genetics
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language:en
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Short-container-title:European Journal of Medical Genetics
Author:
Köse EnginORCID,
Kasapkara Çiğdem Seher,
İnci Aslı,
Yıldız Yılmaz,
Sürücü Kara İlknur,
Kahraman Ayça Burcu,
Tümer Leyla,
Dursun Ali,
Eminoğlu Fatma Tuba
Reference22 articles.
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2. Enzyme replacement therapy for alpha-mannosidosis: 12 months follow-up of a single centre, randomised, multiple dose study;Borgwardt;J. Inherit. Metab. Dis.,2013
3. Alpha-mannosidosis: correlation between phenotype, genotype and mutant MAN2B1 subcellular localisation;Borgwardt;Orphanet J. Rare Dis.,2015
4. Efficacy and safety of Velmanase alfa in the treatment of patients with alpha-mannosidosis: results from the core and extension phase analysis of a phase III multicentre, double-blind, randomised, placebo-controlled trial;Borgwardt;J. Inherit. Metab. Dis.,2018
5. Alpha-mannosidosis: therapeutic strategies;Ceccarini;Int. J. Mol. Sci.,2018