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Content archived on 2024-05-23

A systematic and multidisciplinary approach towards understanding and therapy of the inborn lysosomal storage disease alpha-mannosidosis.

Objective

Alpha-mannosidosis is a rare, degenerative and chronic disease with multiple factors contributing to the degrees of progression. Since these patients are born healthy and exhibiting a slow progression of clinical symptoms, an effective therapy initiated at early age would contribute to a normal development. Thus, this Project is undertaken to reach such a therapy. Due to the complexity of the disease, this Project requires an international effort with a plethora of different techniques and disciplines involved. The Project will aim to reach a critical mass of understanding on both the molecular and path physiological level, and to use this knowledge to develop new strategies for therapy, using a mouse model of the disease. Based on these studies a protocol for clinical trials in humans will be designed, and a company within the Partnership will initiate a commercial production of a therapeutic agent.

Call for proposal

Data not available

Coordinator

UNIVERSITY OF TROMSOE
EU contribution
No data
Address

9037 TROMSOE
Norway

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Total cost
No data

Participants (8)