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Funded by an IRDiRC member =

    • AUSTRIA
    • WIEN
    • WIEN
    • Therapeutic challenge in leukodystrophies
    • Medizinische Universität Wien
    • Center for Brain Research
    • More details
    • AUSTRIA
    • WIEN
    • WIEN
    • MYELINET: Myelin orphan diseases in health (coordination)
    • Medizinische Universität Wien
    • Center for Brain Research
    • More details
    • CANADA
    • Ontario
    • OTTAWA
    • Funded by an IRDiRC member
    • Emerging team in rare diseases: achieving the "triple aim" for inborn errors of metabolism
    • Children's Hospital of Eastern Ontario
    • Newborn Screening Ontario
    • More details
    • CANADA
    • Ontario
    • OTTAWA, ONTARIO
    • Funded by an IRDiRC member
    • Emerging team in rare diseases: achieving the "triple aim" for inborn errors of metabolism
    • University of Ottawa
    • Department of Epidemiology and Community Medicine
    • More details
    • CANADA
    • Québec
    • MONTRÉAL
    • Clinical, radiological and genetic characterization of new forms of leukodystrophies
    • Montreal Children's Hospital Research Institute - McGill University
    • Pediatric Neurodegenerative Laboratory
    • More details
    • FRANCE
    • ALSACE
    • STRASBOURG
    • MYELINET: myeline repairing: role of steroids and sex in leukodystrophy
    • Université Louis Pasteur
    • Laboratoire d'Imagerie et de Neurosciences Cognitives
    • More details
    • FRANCE
    • ALSACE
    • STRASBOURG
    • MYELINET: myeline repairing: cellular and gene therapies strategies
    • Université Louis Pasteur
    • Laboratoire d'Imagerie et de Neurosciences Cognitives
    • More details
    • FRANCE
    • ALSACE
    • STRASBOURG
    • MYELINET: myeline repairing: animal models creation of myelin diseases
    • Université Louis Pasteur
    • Laboratoire d'Imagerie et de Neurosciences Cognitives
    • More details
    • FRANCE
    • ALSACE
    • STRASBOURG
    • MYELINET: myeline repairing: noninvasive white matter imaging
    • Université Louis Pasteur
    • Laboratoire d'Imagerie et de Neurosciences Cognitives
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • LE KREMLIN-BICÊTRE
    • MYELINET: myeline repairing: role of steroids and sex in leukodystrophy
    • GHU Paris-Sud - Hôpital de Bicêtre
    • Stéroïdes, neuroprotection et neurorégénération
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • LE KREMLIN-BICÊTRE
    • LEUKOTREAT: innovative gene and cell therapies in leukodystrophies (WP4)
    • GHU Paris-Sud - Hôpital de Bicêtre
    • Service de Neurologie Pédiatrique
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • LE KREMLIN-BICÊTRE
    • Pain impact in children affected by lysosomal diseases
    • GHU Paris-Sud - Hôpital de Bicêtre
    • Unité douleur et Soins Palliatifs
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Evaluation of diagnosis and treatment protocols and long-term follow-up of errors of metabolism
    • CHU Paris - Hôpital Necker-Enfants Malades
    • Unité fonctionnelle métabolisme
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Evaluation of diagnosis and treatment protocols and long-term follow-up of errors of metabolism
    • CHU Paris - Hôpital Robert Debré
    • Service de neurologie pédiatrique et des maladies métaboliques
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Role of Sox17 in the development and regeneration of oligodendrocytes from mouse and humain brain
    • CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
    • Développement, pathologies gliales et réparation
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Preclinical animal model development of the central nervous system demyelinisation, particularly in leukodystrophy
    • CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
    • Développement, pathologies gliales et réparation
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Function of repulsive proteins Slit and their receptors in proliferation, migration and differenciation of SVZ derivated neuronal precursors in normal and demyelinisated central nervous system, particularly in leukodystrophy
    • CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
    • Développement, pathologies gliales et réparation
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Role of the exogenous stem cells in the central nervous system remyelinisation
    • CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
    • Développement, pathologies gliales et réparation
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • LEUKOTREAT: innovative gene and cell therapies in leukodystrophies (WP4)
    • CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
    • Développement, pathologies gliales et réparation
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Molecules of guidance and remyelinisation in the central nervous system - study of leukodystrophy
    • CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
    • Biologie des interactions neurones / glie
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • LEUKOTREAT: ethical impacts of therapeutic challenges in leukodystrophies (WP5)
    • Centre Universitaire des Saints-Pères - Faculté de Médecine
    • Laboratoire d'éthique médicale et médecine légale
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • New mouse model to study the potential regeneration of glia, repairing of myelin and the interaction between oligodendrocytes / Schwann cells in leukodystrophy
    • ENS - Ecole Normale Supérieure de Paris
    • Genetique Moleculaire Du Developpement
    • More details
    • FRANCE
    • ILE-DE-FRANCE
    • PARIS
    • Function of repulsive proteins Slit and their receptors in proliferation, migration and differenciation of SVZ derivated neuronal precursors in normal and demyelinisated central nervous system, particularly in leukodystrophy
    • Institut de la Vision
    • Département Biologie du Développement
    • More details
    • FRANCE
    • MIDI-PYRENEES
    • TOULOUSE
    • Sialidase Neu4: new therapy for lysosomal diseases
    • CHU de Toulouse - Hôpital Purpan
    • Laboratoire de biochimie
    • More details
    • GERMANY
    • Baden-Württemberg
    • TÜBINGEN
    • LEUKONET- Project 4: Phenotypic variability, natural history and progression markers of late-onset leukodystrophies
    • Hertie-Institut für klinische Hirnforschung (HIH)
    • Sektion klinische Neurogenetik
    • More details
    • GERMANY
    • Baden-Württemberg
    • TÜBINGEN
    • LEUKONET- Project 1 : Epidemiology and description of the natural course of metachromatic leukodystrophy and globoid-cell leukodystrophy (Krabbe disease) manifesting during childhood and adolescence
    • Universitätsklinik für Kinder- und Jugendmedizin Tübingen
    • Abteilung für Neuropädiatrie, Entwicklungsneurologie, Sozialpädiatrie
    • More details
    • GERMANY
    • Hamburg
    • HAMBURG
    • LEUKOTREAT: characterizing leukodystrophies for therapies (WP1)
    • UKE - Universitätsklinikum Hamburg-Eppendorf
    • Arbeitsgruppe Degenerative Gehirnkrankheiten
    • More details
    • GERMANY
    • Mecklenburg-Vorpommern
    • ROSTOCK
    • LEUKONET- Project 4: Phenotypic variability, natural history and progression markers of late-onset leukodystrophies
    • Albrecht-Kossel-Institute for Neuroregeneration (AKos)
    • Albrecht-Kossel-Institut für Neurodegeneration
    • More details
    • GERMANY
    • Mecklenburg-Vorpommern
    • ROSTOCK
    • Biomarker for Krabbe Disease (BioKrabbe): An International, multicentre, epidemiological protocol
    • Albrecht-Kossel-Institute for Neuroregeneration (AKos)
    • Albrecht-Kossel-Institut für Neurodegeneration
    • More details
    • GERMANY
    • Niedersachsen
    • GÖTTINGEN
    • Role of the mitochondrial respiratory process in myelinisation and the interaction axons/glial cells
    • Max-Planck-Institut für experimentelle Medizin
    • Abteilung Neurogenetik
    • More details
    • GERMANY
    • Niedersachsen
    • GÖTTINGEN
    • LEUKOTREAT: pharmacological strategies to treat leukodystrophies (WP3)
    • Max-Planck-Institut für experimentelle Medizin
    • Abteilung Neurogenetik
    • More details
    • GERMANY
    • Niedersachsen
    • GÖTTINGEN
    • LEUKONET- Project 2: Characterization of cerebral alterations in children with unclassified and rare classified leukodystrophies using multiparametric magnetic resonance investigations
    • Universitätsmedizin Göttingen
    • Klinik für Kinder- und Jugendmedizin
    • More details
    • GERMANY
    • Niedersachsen
    • GÖTTINGEN
    • LEUKONET- Project 10: Mutational analysis and rescue of arylsulfatase A and galactocerebrosidase activity by protease inhibitors as a novel therapeutic approach for metachromatic leukodystrophy and globoid cell leukodystrophy
    • Universitätsmedizin Göttingen
    • Klinik für Kinder- und Jugendmedizin
    • More details
    • GERMANY
    • Niedersachsen
    • GÖTTINGEN
    • Clinical and molecular characterization of genetically determined unclear white matter disorders
    • Universitätsmedizin Göttingen
    • Klinik für Kinder- und Jugendmedizin
    • More details
    • GERMANY
    • Rheinland-Pfalz
    • MAINZ
    • Role of the secretion of oligodendroglial exosomes in glial cells for the signaling and the protection of neurons
    • Abteilung Molekulare Zellbiologie der Universität Mainz
    • Abteilung Molekulare Zellbiologie
    • More details
    • GERMANY
    • Sachsen
    • WERMSDORF
    • LEUKONET- Project 4: Phenotypic variability, natural history and progression markers of late-onset leukodystrophies
    • Fachkrankenhaus Hubertusburg gGmbH
    • Klinik für Neurologie
    • More details
    • GREECE
    • GREECE
    • HERAKLION
    • Role of the adhesion molecule TAG-1 in the function of glial cells
    • Institute of Molecular Biology and Biotechnology (IMBB)
    • Department of biology
    • More details
    • HUNGARY
    • Észak-Magyarország
    • BUDAPEST
    • Molecular prognostic factors in pediatric diseases
    • Semmelweis University
    • SOTE Pediatric clinic II
    • More details
    • IRELAND
    • County Dublin
    • DUBLIN
    • A prospective study on the numbers of Irish Travellers attending the metabolic clinic in Dublin
    • Our Lady's Children's Hospital
    • National Centre for Medical Genetics
    • More details
    • ITALY
    • CAMPANIA
    • NAPOLI
    • Funded by an IRDiRC member
    • Modulation of Cellular Clearance in Lysosomal Storage Disorders
    • TIGEM - Telethon Institute of Genetics and Medicine
    • Laboratorio di Ricerca
    • More details
    • ITALY
    • LAZIO
    • ROMA
    • LEUKOTREAT: characterizing leukodystrophies for therapies (WP1)
    • IRCCS Ospedale Pediatrico Bambino Gesù
    • Unità di Malattie Neuromuscolari e Neurodegenerative, Laboratorio di Medicina Molecolare
    • More details
    • ITALY
    • LOMBARDIA
    • MILANO
    • Gene/cell therapy approach for metachromatic and globoid leukodystrophies
    • Istituto San Raffaele Telethon per la Terapia Genica - TIGET
    • Istituto San Raffaele Telethon per la Terapia Genica
    • More details
    • ITALY
    • LOMBARDIA
    • MILANO
    • Combined approaches based on gene transfer and neural stem cells (NSC) for Metachromatic leukodystrophy and Globoid cell leukodystrophy
    • Istituto San Raffaele Telethon per la Terapia Genica - TIGET
    • Istituto San Raffaele Telethon per la Terapia Genica
    • More details
    • ITALY
    • LOMBARDIA
    • MILANO
    • Testing the transplantation efficiency of neural stem cells coupled with gene and pharmacological therapy to point multiples sites of the pathology in a mouse model of Krabbe disease
    • Istituto San Raffaele Telethon per la Terapia Genica - TIGET
    • Istituto San Raffaele Telethon per la Terapia Genica
    • More details
    • ITALY
    • LOMBARDIA
    • MILANO
    • Funded by an IRDiRC member
    • Hsc-based ex vivo gene therapy of metachromatic and globoid leukodystrophy
    • Istituto San Raffaele Telethon per la Terapia Genica - TIGET
    • Unità di Ricerca Clinica Pediatrica
    • More details
    • ITALY
    • LOMBARDIA
    • MILANO
    • LEUKOTREAT: innovative gene and cell therapies in leukodystrophies (WP4)
    • Istituto San Raffaele Telethon per la Terapia Genica - TIGET
    • Unità di Ricerca Clinica Pediatrica
    • More details
    • ITALY
    • LOMBARDIA
    • MILANO
    • Cellule staminali ematopoietiche per la terapia genica nel trattamento della leucodistrofia globoide
    • Istituto San Raffaele Telethon per la Terapia Genica - TIGET
    • Unità di Ricerca Clinica Pediatrica
    • More details
    • ITALY
    • TOSCANA
    • SIENA
    • Krabbe disease: pathogenetic aspects and progress towards gene therapy
    • Azienda Ospedaliera Universitaria Senese - Ospedale Santa Maria alle Scotte
    • Laboratorio di Genetica
    • More details
    • LEBANON
    • Beyrouth
    • BEIRUT
    • Identification et localisation du gène responsable d'une forme de leucodystrophie et d'oligodontie dans une grande famille
    • Université Saint-Joseph
    • Unité de génétique médicale
    • More details
    • PORTUGAL
    • NORTE
    • PORTO
    • Splicing therapeutics for patients affected by lisosomal storage disorders.
    • Instituto Nacional de Saúde Dr. Ricardo Jorge - Porto
    • Unidade de Investigação & Desenvolvimento; Departamento de Genética
    • More details
    • PORTUGAL
    • NORTE
    • PORTO
    • The crosstalk between lipid antigen presentation and the pathogenic mechanisms of Lysosomal Storage Diseases
    • Instituto de Biologia Molecular e Celular
    • Unidade de Biologia do Lisossoma e do Peroxissoma
    • More details
    • SPAIN
    • Cataluña
    • L'HOSPITALET DE LLOBREGAT
    • LEUKOTREAT: biomarkers for leukodystrophies treatment (WP2)
    • IDIBELL - Instituto de Investigación Biomédica de Bellvitge
    • Laboratorio de Enfermedades Neurometabólicas
    • More details
    • UNITED KINGDOM
    • Cambridgeshire
    • CAMBRIDGE
    • EUCLYD: substrate reduction therapy for glycospingolipidoses and mucopolysaccharidoses (WP4)
    • Addenbrooke's Hospital
    • Lysosomal Disorders Unit
    • More details
    • UNITED KINGDOM
    • Cambridgeshire
    • CAMBRIDGE
    • Promoting the remyelinisation in aged central nervous system
    • Cambridge Veterinary School
    • Department of Veterinary Medicine
    • More details
    • UNITED KINGDOM
    • Cambridgeshire
    • CAMBRIDGE
    • Funded by an IRDiRC member
    • NIHR BioResource - Rare Disease: SPEED - Specialist Pathology: Evaluating Exomes in Diagnostics
    • Wellcome Trust/MRC Building
    • Cambridge Institute for Medical Research
    • More details
    • UNITED KINGDOM
    • Greater London
    • LONDON
    • Development of diagnostic methods for lysosomal storage diseases
    • UCL Institute of Child Health, University College London
    • Biochemistry Research Group
    • More details
    • UNITED KINGDOM
    • Lothian
    • EDINBURGH
    • Genetic disorders of human brain development
    • Western General Hospital
    • MRC Human Genetics Unit
    • More details
    • UNITED KINGDOM
    • West Midlands
    • BIRMINGHAM
    • NIPSIGEN - Clinical translation of non-invasive prenatal diagnosis (NIPD) for single gene disorders (Health Innovation Challenge Fund)
    • Birmingham Women's NHS Foundation Trust
    • Regional Genetics Unit
    • More details