Objective 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 are born healthy, an early initiated therapy could contribute to a normal development. To develop an efficient therapy for this disease the collaborative research project EURAMAN and HUE-MAN were initiated within FP5 and FP6, respectively. Within these collaborative networks, European scientists, clinicians and the industry successfully i) developed an efficient pre-clinical Enzyme Replacement Therapy (ERT) protocol using recombinant human (rh) LAMAN in a mouse model for alpha-Mannosidosis, ii) built up a database collecting patient data and iii) defined clinical endpoints for the future clinical trials in alpha-Mannosidosis patients by an European wide natural history study. Furthermore, the HUE-MAN network developed the conditions for a large-scale production of the recombinant enzyme and the way is now paved for the first clinical trials in man, which we aim to realize within FP7. The main objectives of the ALPHA-MAN project are i) the performance of efficient clinical trials (phase I-III) in alpha-Mannosidosis patients, ii) a better understanding of the pathophysiology and the mechanism of how the recombinant enzyme enters the cells of the central nervous system by performing ERT in a newly generated immuntolerant alpha-Mannosidosis mouse model, which allows chronic treatment and iii) the determination of the minimal effective dose with chronic treatment in the immuntolerant mice. Fields of science natural sciencesbiological sciencesneurobiologynatural sciencescomputer and information sciencesdatabaseshumanitieshistory and archaeologyhistorymedical and health sciencesbasic medicinephysiologypathophysiologynatural sciencesbiological sciencesbiochemistrybiomoleculesproteinsenzymes Programme(s) FP7-HEALTH - Specific Programme "Cooperation": Health Topic(s) HEALTH.2010.2.4.4-1 - Clinical development of substances with a clear potential as orphan drugs. FP7-HEALTH-2010-single-stage Call for proposal FP7-HEALTH-2010-single-stage See other projects for this call Funding Scheme CP-FP - Small or medium-scale focused research project Coordinator CHRISTIAN-ALBRECHTS-UNIVERSITAET ZU KIEL Address Olshausenstrasse 40 24118 Kiel Germany See on map Region Schleswig-Holstein Schleswig-Holstein Kiel, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Administrative Contact Paul Saftig (Prof.) Links Contact the organisation Opens in new window Website Opens in new window EU contribution No data Participants (11) Sort alphabetically Sort by EU Contribution Expand all Collapse all Zymenex AS Denmark EU contribution € 329 732,00 Address Roskildevej 12c 3400 Hillerød See on map Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Jens Fogh (Dr.) Links Contact the organisation Opens in new window Other funding No data REGION HOVEDSTADEN Denmark EU contribution € 2 826 623,00 Address Kongens vaenge 2 3400 Hillerod See on map Region Danmark Hovedstaden Nordsjælland Activity type Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments) Administrative Contact Allan Meldgaard Lund (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data KATHOLIEKE UNIVERSITEIT LEUVEN Belgium EU contribution € 299 360,00 Address Oude markt 13 3000 Leuven See on map Region Vlaams Gewest Prov. Vlaams-Brabant Arr. Leuven Activity type Higher or Secondary Education Establishments Administrative Contact Elke Lammertyn (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data THE UNIVERSITY OF MANCHESTER United Kingdom EU contribution € 37 282,00 Address Oxford road M13 9PL Manchester See on map Region North West (England) Greater Manchester Manchester Activity type Higher or Secondary Education Establishments Administrative Contact Liz Fay (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data UNIVERSITAETSMEDIZIN DER JOHANNES GUTENBERG-UNIVERSITAET MAINZ Germany EU contribution € 589 434,00 Address Langenbeckstrasse 1 55131 Mainz See on map Region Rheinland-Pfalz Rheinhessen-Pfalz Mainz, Kreisfreie Stadt Activity type Higher or Secondary Education Establishments Administrative Contact Silvia Tschauder (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data HOSPICES CIVILS DE LYON France EU contribution € 255 561,00 Address Quai des celestins 3 69002 Lyon See on map Region Auvergne-Rhône-Alpes Rhône-Alpes Rhône Activity type Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments) Administrative Contact Cecile Legros (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data UNIVERSITETET I TROMSOE - NORGES ARKTISKE UNIVERSITET Norway EU contribution € 97 400,00 Address Hansine hansens veg 14 9019 Tromso See on map Region Norge Nord-Norge Troms og Finnmark Activity type Higher or Secondary Education Establishments Administrative Contact Stig Orsje (Mr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data TEKNOLOGISK INSTITUT Denmark EU contribution € 187 636,00 Address Gregersensvej 1 2630 Taastrup See on map Region Danmark Hovedstaden Københavns omegn Activity type Research Organisations Administrative Contact Anne Maria Hansen (Ms.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data INSTYTUT POMNIK CENTRUM ZDROWIA DZIECKA Poland EU contribution € 11 172,00 Address Aleja dzieci polskich 20 04730 Warszawa See on map Region Makroregion województwo mazowieckie Warszawski stołeczny Miasto Warszawa Activity type Research Organisations Administrative Contact Anna Tylki-Szymańska (Prof.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data LARIX AS Denmark EU contribution € 543 958,00 Address Tempovej 44 - 1st floor 2750 Ballerup See on map Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Klaus Juel Olsen (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data UNILABAS AS Denmark EU contribution € 115 864,00 Address Nygaardsvej 32 2100 Copenhagen See on map Activity type Private for-profit entities (excluding Higher or Secondary Education Establishments) Administrative Contact Persson Poul (Dr.) Links Contact the organisation Opens in new window Website Opens in new window Other funding No data
