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AMFAs are non\u2010phosphate serum\u2010resistant derivatives that efficiently bind the cation\u2010independent mannose 6\u2010phosphate receptor (CI\u2010M6PR), which is the main pathway to address enzymes to lysosomes. One of the AMFAs was used to improve the treatment of the lysosomal myopathy Pompe disease, in which acid \u03b1\u2010glucosidase (GAA) is defective. AMFA grafting on a M6P\u2010free recombinant GAA led to a higher uptake of the GAA in adult Pompe fibroblasts in culture as compared to Myozyme, the M6P recombinant GAA. Moreover, the treatment of Pompe adult mice with the AMFA\u2010grafted recombinant enzyme led to a remarkable improvement, even at low doses, in muscle functionality and regeneration, whereas Myozyme had limited efficacy.<\/jats:p>","DOI":"10.1002\/anie.201607824","type":"journal-article","created":{"date-parts":[[2016,10,24]],"date-time":"2016-10-24T04:07:52Z","timestamp":1477282072000},"page":"14774-14777","source":"Crossref","is-referenced-by-count":27,"title":["Design of Potent Mannose 6\u2010Phosphate Analogues for the Functionalization of Lysosomal Enzymes To Improve the Treatment of Pompe Disease"],"prefix":"10.1002","volume":"55","author":[{"given":"Khaled","family":"El\u2005Cheikh","sequence":"first","affiliation":[{"name":"NanoMedSyn  34093 Montpellier cedex 05 France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilaria","family":"Basile","sequence":"additional","affiliation":[{"name":"NanoMedSyn  34093 Montpellier cedex 05 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