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Clinical development of Enzyme Replacement Therapy in alpha-Mannosidosis patients using recombinant human enzyme (ALPHA-MAN)
Date du début: 1 oct. 2010, Date de fin: 31 mars 2014,
Alpha-Mannosidosis is a rare Lysosomal Storage Disorder with a worldwide incidence of about 1:500 000. This orphan and devastating disease is caused by the deficiency of the lysosomal alpha-mannosidase (LAMAN) which is responsible for the intralysosomal degradation of mannosyl linked oligosaccharides. To date no causative treatment for alpha-Mannosidosis is available and since most of the children ...
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