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Exciting new data regarding positive uptake and effect of recombinant human Alpha-mannosidase enzyme in Alpha-mannosidosis mice* from the EU sponsored Alpha-mannosidosis Hue-Man consortium group, were presented at the Hue-Man meeting, which took place in Prague, Czech republic, during the days of October 31 - November 2, 2008. The data demonstrated a reversal of peripheral and central neural storage and ataxia in the mice after enzyme replacement therapy. The natural history study of the human disease in Alpha-mannosidosis patients is ongoing and recruitment and planned time point evaluations are being performed as scheduled, in order to define clinical endpoints for the upcoming first clinical trials in man.
 

*   Reversal of Peripheral and Central Neural Storage and Ataxia after Recombinant Enzyme Replacement Therapy in alpha-Mannosidosis Mice: Blanz J, Stroobants S, Lüllmann-Rauch R, Morelle W, Lüdemann M, D'Hooge R, Reuterwall H, Michalski JC, Fogh J, Andersson C and Saftig P. Human Molecular Genetics, 2008, Vol. 17, No. 22: 3437 - 3445

About the disease

The disease is due to a deficiency of the a-mannosidase enzyme and affects approximately 500 patients worldwide. Alpha-mannosidosis is a rare inborn disorder that results in mental retardation, skeletal changes, hearing loss, recurrent infections and progression to early death. The children are often born apparently normal and their conditions worsen progressively, without any possibility to prevent this evolution. In the children that are born healthy, a therapy initiated at an early age could contribute to a normal development. Today, the most promising therapy for lysosomal storage diseases is enzyme replacement therapy (ERT), here the enzyme lacking in the patient is introduced into the blood stream, from where it is internalized by the cells and reaches the lysosomes, acting as the original missing enzyme.

About the Hue-Man consortium

The HUE-MAN partnership www.uni-kiel.de/Biochemie/hue-man consists of scientists and clinicians from: the Christian Albrechts University, Kiel, Germany; the Katholieke Universiteit, Leuven, Belgium; the Georg August University, Göttingen, Germany; the Central Manchester & Manchester Children's Hospitals NHS Trust, Manchester, United Kingdom; the Johannes Gutenberg University, Mainz, Germany; the Charles University, Prague, Czech Republic; the University of Tromsoe, Tromsoe, Norway; the Centre National de la Researche Scientific (CNRS), Lille, France and Zymenex.

Further information

CEO Jens Fogh, Zymenex A/S, Roskildevej 12 C, Hillerød, Denmark, telephone +45 48 25 00 54