Enzyme replacement therapy with velmanase alfa (human recombinant alpha-mannosidase): Novel global treatment response model and outcomes in patients with alpha-mannosidosis.
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D. Ardigò | C. Hendriksz | A. Lund | P. Harmatz | N. Guffon | J. Hennermann | F. Cattaneo | S. Geraci | L. Borgwardt | Silvia Geraci