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Research projects

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

BELGIUM

ANTWERPEN
ANTWERPEN

CANADA

Alberta
EDMONTON

Funded by an IRDiRC memberMRI biomarkers in ALS
University of Alberta
Department of Medicine, Division of Neurology

CANADA

Alberta
EDMONTON

Funded by an IRDiRC memberPromoting protection of functionally intact motor units in amyotrophic lateral sclerosis (ALS).
University of Alberta
Faculty of Physical Education and Recreation

CANADA

Colombie-Britannique
VANCOUVER

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberLoss of VAPB Function in Amyotrophic Lateral Sclerosis-8 (ALS8)
Ottawa Hospital Research Institute
Neuroscience

CANADA

Ontario
TORONTO

Funded by an IRDiRC memberThe TAR DNA-Binding Protein (TDP-43) and Amyotrophic Lateral Sclerosis
University of Toronto
Centre for Research in Neurodegenerative Disease

CANADA

Ontario
WATERLOO

Funded by an IRDiRC memberFolding and aggregation of ALS-associated mutant superoxide dismutases
University of Waterloo
Department of Chemistry

CANADA

Québec
MONTRÉAL

Funded by an IRDiRC memberRegeneration of motor neurons controlling movement and respiration from embryonic stem cells
Montreal Neurological Institute and Hospital
McGill University

CANADA

Québec
MONTRÉAL

Funded by an IRDiRC memberRegulation of motor neuron identity and circuit development
Montreal Neurological Institute and Hospital
McGill University

FINLAND

Finland
HELSINKI

Funded by an IRDiRC memberCDNF and CDNF variants- novel therapy for ALS
Biocenter 1, University of Helsinki
Institute of Biotechnology

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

Funded by an IRDiRC memberGenetics of Young Amyotrophic Lateral Sclerosis
Institution: Information not provided - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

BOURGOGNE-FRANCHE-COMTE
DIJON

Funded by an IRDiRC memberDEMENTIA : Exploiting genetic biomarkers of neurodegenerative diseases for developing early diagnostic tests and possible treatments - FR
Faculté des Sciences de Santé
Institut de Chimie Moléculaire de l'Université de Bourgogne (ICMUB) - CNRS 6302

FRANCE

CENTRE-VAL DE LOIRE
ORLÉANS

Evaluation of new inhibitors of LIMK1 and LIMK2, kinases of the Rho-GTPase pathway, in a mouse model of amyotrophic lateral sclerosis
UPR4301 Centre de biophysique moléculaire (CBM)
Equipe signalisation cellulaire et neurofibromatose

FRANCE

CENTRE-VAL DE LOIRE
TOURS

SUMOylation and aggregation of TDP43: roles in the pathophysiology of amyotrophic lateral sclerosis
CHRU de Tours - Hôpital Bretonneau
Laboratoire de biochimie et biologie moléculaire

FRANCE

CENTRE-VAL DE LOIRE
TOURS CEDEX 1

New therapeutic strategy in ALS based on metabolic status and associated metabolic disorders
Université de Tours
Imagerie et Cerveau "Neurogénétique et Neurométabolome" Equipe 2

FRANCE

GRAND-EST
ILLKIRCH-GRAFFENSTADEN

Therapeutic approach for Amyotrophic Lateral Sclerosis due to G4C2 expansion in C9ORF72
Institut de génétique et de biologie moléculaire et cellulaire - IGBMC
Département Médecine translationnelle et neurogénétique

FRANCE

GRAND-EST
ILLKIRCH-GRAFFENSTADEN

Funded by an IRDiRC memberC9ORF72-ALS : Role of C9ORF72 in Amyotrophic Lateral Sclerosis - FR
Institut de génétique et de biologie moléculaire et cellulaire - IGBMC
Département Médecine translationnelle et neurogénétique

FRANCE

GRAND-EST
STRASBOURG

Involvement of metabolic dysfunctions in the pathogenesis of amyotrophic lateral sclerosis
Faculté de médecine de Strasbourg - Louis Pasteur
Laboratoire de signalisations moléculaires et neurodégénérescence

FRANCE

GRAND-EST
STRASBOURG

Mechanisms of the spread of FUS proteininopathy during amyotrophic lateral sclerosis
Faculté de médecine de Strasbourg - Louis Pasteur
Mécanismes centraux et périphériques de la Neurodégénérescence - INSERM U1118

FRANCE

GRAND-EST
STRASBOURG

Funded by an IRDiRC memberToFU: Interactions between TAU, FUS and TDP-43 in neurodegenerative diseases - FR
Faculté de médecine de Strasbourg - Louis Pasteur
Mécanismes centraux et périphériques de la Neurodégénérescence - INSERM U1118

FRANCE

GRAND-EST
STRASBOURG

Funded by an IRDiRC memberMAXOMOD: Multi-omic analysis of axono-synaptic degeneration in motoneuron disease - FR
Institut Pluridisciplinaire Hubert Curien
Spectrométrie de masse bio organique

FRANCE

GRAND-EST
VANDOEUVRE LES NANCY CEDEX

FRANCE

HAUTS-DE-FRANCE
LILLE

FRANCE

ILE-DE-FRANCE
EVRY

FRANCE

ILE-DE-FRANCE
PARIS

The issue of prenatal / preimplantation diagnosis in parentage information in neurogenetic and neuromuscular dominant diseases
CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
UF de Neurogénétique Moléculaire et Cellulaire

FRANCE

ILE-DE-FRANCE
PARIS

Presymptomatic neuromuscular junction defects and compensatory mechanisms in ALS
CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
Unité Nerf périphérique, Sclérose Latérale Amyotrophique (SLA)

FRANCE

ILE-DE-FRANCE
PARIS

Involvement of metabolic dysfunctions in the pathogenesis of amyotrophic lateral sclerosis
CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
Département de Neurologie - Pôle des Maladies du Système Nerveux

FRANCE

ILE-DE-FRANCE
PARIS

From Movement Preparation to Gait Execution in ALS: : Combined Neurophysiological and Neuroimaging Approaches - FR
CHU Paris-GH La Pitié Salpêtrière-Charles Foix - Hôpital Pitié-Salpêtrière
Groupement hospitalier universitaire Pitié-Salpétrière

FRANCE

ILE-DE-FRANCE
PARIS

First study in France searching a link between Amyotrophic Lateral Sclerosis (ALS) and beta-N-methylamino-L-alanine (BMAA) cyanotoxin
Hôpitaux Paris Centre - site Cochin
Centre de Références des Maladies Neuromusculaires (CRMN)

FRANCE

ILE-DE-FRANCE
PARIS

New gene therapy for C9ORF72-linked ALS-FTD
Institut de Myologie - Hôpital Pitié-Salpêtrière
Centre de Recherche en Myologie

FRANCE

ILE-DE-FRANCE
PARIS

Peripheral macrophages as a promising target to modulate ALS disease progression
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Sclérose latérale amyotrophique : causes et mécanismes de la dégénérescence des neurones moteurs"

FRANCE

ILE-DE-FRANCE
PARIS

Gene hunting in familial Amyotrophic Lateral Sclerosis
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Sclérose latérale amyotrophique : causes et mécanismes de la dégénérescence des neurones moteurs"

FRANCE

ILE-DE-FRANCE
PARIS

Study of the interactions between microglial cells, macrophages and neurons from three axes in an animal model of amyotrophic lateral sclerosis
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Bases moléculaires, physiopathologie et traitement des maladies neurodégénératives"

FRANCE

ILE-DE-FRANCE
PARIS

Implication of peripheral macrophages in ALS
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Bases moléculaires, physiopathologie et traitement des maladies neurodégénératives"

FRANCE

ILE-DE-FRANCE
PARIS

Localisation and identification of causative genes for amyotrohic lateral sclerosis
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Excitabilité, synapse & pathologies associées"

FRANCE

ILE-DE-FRANCE
PARIS

Study of the pathological consequences of the loss of function of the C9ORF72 gene and its implication in autophagy
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Traitement de la sclérose latérale amyotrophique : de la génétique au poisson zèbre"

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberPredict to prevent frontotemporal lobar degeneration (FTLD) and amyotrophic lateral sclerosis (ALS)
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Institut du Cerveau et de la Moëlle épinière - INSERM U1127

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberHUMAn Neuromuscular Integrative System for drug discovery
Université Paris Descartes - Paris 5
Centre de Neurophysique, Physiologie et Pathologie - CNRS UMR 8119

FRANCE

ILE-DE-FRANCE
PARIS

Can a spinal interneuropathy contribute to motoneuron degeneration in ALS? An electrophysiological study on patients and mouse models of ALS
Université Paris Descartes - Paris 5
Centre de Neurophysique, Physiologie et Pathologie - CNRS UMR 8119

FRANCE

ILE-DE-FRANCE
PARIS CEDEX 06

Is spinal motoneuron degeneration in ALS caused by their abnormal excitability?
Centre de Neurophysique, Physiologie et Pathologie - CNRS UMR 8119
Spinal physiology and pathophysiology

FRANCE

ILE-DE-FRANCE
POISSY

Identification and characterization of gene mutations in patients with azoospermia by maturation
Centre hospitalier intercommunal Poissy-Saint-Germain-en-Laye
Unité de Cytogénétique

FRANCE

NOUVELLE AQUITAINE
BORDEAUX

Study of the Expression of Micro-RNA as Biomarker Diagnosis and Prognosis in Amyotrophic Lateral Sclerosis- FR
CHU - Hôpitaux de Bordeaux
Direction de la Recherche Clinique et de l'Innovation (DRCI) - Hôpitaux de Bordeaux

FRANCE

NOUVELLE AQUITAINE
BORDEAUX CEDEX

Bumetanide treatment of the early alteration of chloride homeostasis in ALS mouse spinal motoneurons
Institut de Neurosciences cognitives et intégratives d'Aquitaine INCIA
Développement des réseaux neuronaux en conditions normales et pathologiques

FRANCE

NOUVELLE AQUITAINE
LIMOGES CEDEX

FRANCE

OCCITANIE
MONTPELLIER

ALS in Herault: epidemiology ans exposure into and outside the thau cluster
CHU de Montpellier - Hôpital Gui de Chauliac
Unité Scléroses en plaques et substances blanches

FRANCE

OCCITANIE
MONTPELLIER CEDEX 05

thwart TDP-43-induced mitochondrial defects in amyotrophic lateral sclerosis
Mécanismes moléculaires dans les démences neurodégénératives (MMDN)
Equipe 4 : La maladie de Huntington : neurophysiopathologie de la drosophile à la souris

FRANCE

OCCITANIE
MONTPELLIER CEDEX 5

FRANCE

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

FRANCE

PROVENCE-ALPES-COTE D'AZUR
MARSEILLE

GERMANY

Baden-Württemberg
TÜBINGEN

GERMANY

Baden-Württemberg
TÜBINGEN

MITOTARGET: in vivo evidence of mitochondrial-targeted therapeutics (WP5)
Institut für Medizinische Genetik und angewandte Genomik Tübingen
Institut für Medizinische Genetik und angewandte Genomik

GERMANY

Baden-Württemberg
ULM

Epidemiology of motoneuron diseases - A case-control study
Universitäts- und Rehabilitationskliniken Ulm (RKU)
Klinik für Neurologie

GERMANY

Baden-Württemberg
ULM

GERMANY

Bayern
WÜRZBURG

Funded by an IRDiRC memberCALSER : The effect of CDNF in ALS and ER stress (partner no 2)
Institut für Klinische Neurobiologie der Universität Würzburg
Institut für Klinische Neurobiologie

GERMANY

Berlin
ADDRESS: NOT PROVIDED - DE

Funded by an IRDiRC memberMAIV: Modelling of ALS by direct reprogramming
Institution: Information not provided - DE

GERMANY

Hessen
FRANKFURT AM MAIN

MITOTARGET: Mitochondriale Membran-Eigenschaften im Prozess der Neurodegeneration und Untersuchung der Effekte von neuen kleinen Molekülen (WP2)
Johann Wolfgang Goethe-Universität Frankfurt
Pharmakologisches Institut für Naturwissenschaftler

GERMANY

Nordrhein-Westfalen
BONN

Role of PGC-1A in the evolution of amyotrophic lateral sclerosis and effects of its regulation
Universitätsklinikum Bonn (AöR)
Klinik für Neurodegenerative Erkrankungen & Gerontopsychiatrie

GERMANY

Nordrhein-Westfalen
MÜNSTER

GERMANY

Sachsen
DRESDEN

Funded by an IRDiRC memberCureALS: Stress granules and proteostasis in motor neurons: towards a mechanistic understanding of ALS (coordination)
Max Planck Institute of Molecular Cell Biology and Genetics
Forschungsgruppe Alberti

IRELAND

County Dublin
DUBLIN

Funded by an IRDiRC memberBRAINMEND - Biological Resource Analysis to Identify New Mechanisms and phenotypes in Neurodegenerative Diseases - IE
Trinity College Dublin
Department of Neurology - Trinity Biomedical Sciences Institute

IRELAND

County Dublin
DUBLIN

IRELAND

County Dublin
DUBLIN

IRELAND

County Dublin
DUBLIN

ISRAEL

ISRAEL
ADDRESS: NOT PROVIDED - IL

ISRAEL

ISRAEL
TEL AVIV

Funded by an IRDiRC memberALS-degeneration: The molecular basis for neurodegeneration and muscle atrophy in ALS - IL
University of Tel-Aviv
Dept. of Physiology and Pharmacology

ITALY

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

Funded by an IRDiRC memberUncovering the function of HDAC4 in skeletal muscle
Institution: Information not provided - IT

ITALY

LAZIO
ROMA

Proposal for an integrated approach to rare diseases: a study between basic laboratory models and clinical epidemiology in amyotrophic lateral sclerosis (ALS)
ISS - Istituto Superiore di Sanità
Reparto Farmaci per la Salute del Bambino e della Donna - Dipartimento del Farmaco

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberExosomal RNA for the diagnosis of ALS
ISS - Istituto Superiore di Sanità
Centro Nazionale per la Ricerca e la Valutazione preclinica e clinica dei Farmaci

ITALY

LOMBARDIA
MILANO

Modulating motor neuron vulnerability using the oculomotor restricted genes IGF-2 and SYT13 for ALS therapy
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Dipartimento di Scienze Neurologiche

ITALY

LOMBARDIA
MILANO

Preclinical studies aimed to develop target genes based therapies for the treatment of amyotrophic lateral sclerosis
Istituto di Ricerche Farmacologiche "Mario Negri"
Laboratorio di Neurobiologia Molecolare

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberNovel biomarkers of amyotrophic lateral sclerosis: role in pathogenesis and possible therapeutic targets
Istituto di Ricerche Farmacologiche "Mario Negri"
Laboratorio di Proteomica Traslazionale

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberNEUROMICS: Integrated European Project on Omics Research of Rare Neuromuscular and Neurodegenerative Diseases - IT
Università degli Studi di Milano - Scienze Farmacologiche e Biomolecolari
Laboratorio di Biologia delle Cellule Staminali

ITALY

LOMBARDIA
PAVIA

Novel therapeutic strategies for Spinal Muscular Atrophy: testing the efficacy of cell-permeable peptides in preclinical models
Fondazione Salvatore Maugeri
Laboratorio di Ricerca sulle malattie Neurodegenerative

ITALY

PIEMONTE
TORINO

Funded by an IRDiRC memberMutations in the MATR3 gene as a cause of amyotrophic lateral sclerosis (ALS)
Ospedale Molinette
Centro regionale esperto per la sclerosi laterale amiotrofica

ITALY

PIEMONTE
TORINO

ITALY

PUGLIA
BARI

Amyotrophic lateral sclerosis (SLA) and riboflavin responsive multiple Acyl-CoA dehydrogenase deficiency (RR-MADD)
Università degli Studi di Bari Aldo Moro
Laboratorio di Biochimica e Biologia Molecolare E.Quagliarello

JAPAN

JAPAN
ADDRESS : NOT PROVIDED - JP

JAPAN

JAPAN
ADDRESS : NOT PROVIDED - JP

Funded by an IRDiRC memberElucidation of molecular bases of intractable neurological diseases
Institution: Information not provided - JP

JAPAN

JAPAN
OSAKA

JAPAN

JAPAN
OSAKA

NETHERLANDS

Zuid-Holland
LEIDEN

PORTUGAL

SUL
LISBOA

Respiratory Rehabilitation in Amyotrophic Lateral Sclerosis: clinical and biochemical impact.
Faculdade de Medicina da Universidade de Lisboa
Unidade de Neuromusculares

PORTUGAL

SUL
OEIRAS

Mechanisms of SOD1 toxic aggregation in neurodegenerative processes
Instituto de Tecnologia Química e Biológica
Protein Biochemistry Folding and Stability

SPAIN

Aragón
ZARAGOZA

Circular RNAs: New modulators of gene expression in Amyotrophic Lateral Sclerosis
Universidad de Zaragoza. Facultad de Veterinaria
Departamento de Anatomía, Embriología y Genética Animal

SPAIN

Castilla - La Mancha
TOLEDO

A potential metabolic-targeted therapeutic for Amyotrophic lateral sclerosis (ALS): Leptin
Hospital Nacional de Parapléjicos
Laboratorio de Neurometabolismo

SPAIN

Madrid
MADRID

Relationship Between Neurological Disability and Visual Impairment in Patients With ALS or Friedreich's Ataxia - ES
Hospital General Universitario Gregorio Marañón
Servicio de Oftalmología

SPAIN

Murcia
SANTA LUCÍA

SWEDEN

Stockholms läns landsting
STOCKHOLM

In vitro molecular mechanism of amyotrophic lateral sclerosis
Stockholm University
Department of Biochemistry and Biophysics

SWEDEN

Västerbottens läns landsting
UMEÅ

SWEDEN

Västerbottens läns landsting
UMEÅ

Studies of misfolded SOD1 in amyotrophic lateral sclerosis (ALS)
Umeå University
Department of Medical Biosciences

SWEDEN

Västerbottens läns landsting
UMEÅ

Molecular portfolio of the extraocular muscles: clues to protection in muscular disease
University Hospital of Umeå
Department of Clinical Sciences

SWEDEN

Västerbottens läns landsting
UMEÅ

Genetic factors causing sporadic and familial amyotrophic lateral sclerosis (ALS/MND) with or without dementia
University Hospital of Umeå
Department of Pharmacology and Clinical Neuroscience

UNITED KINGDOM

Edinburgh
EDINBURGH

Funded by an IRDiRC memberCellular and molecular insights into neurodegeneration mediated by the C9orf72 gene mutation amyotrophic lateral sclerosis
University of Edinburgh
Centre for Clinical Brain Sciences, Chancellor's Building

UNITED KINGDOM

Greater London
LONDON

UNITED KINGDOM

Greater London
LONDON

Funded by an IRDiRC memberInvestigating deficits of axonal RNA metabolism and axonal signalling in amyotrophic lateral sclerosis
UCL Institute of Neurology, University College London,
Clinical Neurosciences

UNITED KINGDOM

Greater London
LONDON

Funded by an IRDiRC memberInvestigating neuronal RNA localisation and translational deficits as gain of function mechanisms in ALS.
UCL Institute of Neurology, University College London,
Clinical Neurosciences

UNITED KINGDOM

Greater London
LONDON

UNITED KINGDOM

Greater Manchester
ADDRESS: NOT PROVIDED - UK

UNITED KINGDOM

Oxfordshire
HEADINGTON

UNITED KINGDOM

Oxfordshire
OXFORD

Identifying effectors of mutant C9Orf72 ALS/FTD to combat neurodegeneration
Le Gros Clark Building - University of Oxford
Neuroscience

UNITED KINGDOM

Oxfordshire
OXFORD

Funded by an IRDiRC memberAntisense Oligonucleotide Therapy for Neuromuscular Disease
University of Oxford
Department of Physiology, Anatomy and Genetics

UNITED KINGDOM

South Yorkshire
SHEFFIELD

Investigating the role of exosomal RNA in C9orf72 astrocyte toxicity - moving towards gene therapy approaches
Sheffield Institute of Translational Neuroscience, University of Sheffield
Department of Neuroscience

UNITED KINGDOM

Tyne & Wear
NEWCASTLE UPON TYNE

TREAT-NMD: Accelerating Treatments for Neuromuscular Diseases (coordination)
Newcastle upon Tyne Hospitals NHS Trust
John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine

UNITED STATES

Massachusetts
BOSTON

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMisfolded als-linked profilin-1: a novel therapeutic target
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberC9orf72 in motor system biology and als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberTdp-43 and mitochondrial dysfunction in als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberModeling the progression of sod1-linked motor neuron disease
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberExtrinsic mechanisms governing injury-induced axon degeneration
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberInvestigating disease mechanisms in c9orf72-linked als/ftd
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberP97 inhibitors for treatment of ibmpfd/als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMuscle tregs in health and disease
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMechanisms of neurodegeneration in als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberAmpk; metabolism and als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberEpigenetic editing of mutant c9orf72
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberInvestigating a2-chimaerin-dependent motor neuron protection in als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberInvestigation of als caused by mutant chchd10
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMechanisms of hipk2 in neurodegeneration
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberNucleocytoplasmic transport and nuclear pore disruption in als/ftd
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRole of c9orf72 in neurodegeneration
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRole of oligodendroglia in the pathogenesis of als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberStudy on hnrnpa1 pathobiology in als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberUnderstanding sod1 kinetics in amyotrophic lateral sclerosis
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberDetermination of cell death pathways activated in als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberDrosophila models for ubiquilin-associated als
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberOligodendroglial dysfunction in c9orf72 als and ftd
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRetromer dysfunction in amyotrophic lateral sclerosis
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberStudy on neurodegeneration using tdp-43 transgenic rats
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberThe nuclear pore complex in als/ftd
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberFg-nucleoporins and nuclear transport disruption in c9orf72-als/ftd
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMechanisms of motor neuron disease
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberNad metabolsim and mitochondrial dysfunction in als models
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRedesign rat model for als research
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRegulation of glutamate transport in astrocyte subtypes and in als
Institution: Information not provided - US

CANADA

Ontario
OTTAWA

CANADA

Ontario
OTTAWA

Funded by an IRDiRC memberThe molecular pathogenesis of spinal muscular atrophy
Ottawa Hospital Research Institute
Regenerative Medicine

CANADA

Ontario
OTTAWA, ONTARIO

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCE

ILE-DE-FRANCE
EVRY

Funded by an IRDiRC memberSEDUCE : Stem cells for drug screening targeting splices - FR
GENOPOLE - Campus 1
I-Stem - Institut des cellules Souches pour le Traitement et l'Etude des maladies Monogéniques

FRANCE

ILE-DE-FRANCE
PARIS

FRANCE

OCCITANIE
MONTPELLIER

Role of AnxA2 mRNA axonal trafficking in Spinal Muscular Atrophy (SMA)
UMR5535 Institut de génétique moléculaire de Montpellier (IGMM)
Assemblage et Trafic de Ribonucléoprotéines

GERMANY

Baden-Württemberg
FREIBURG

SMArtCARE : Clinical evaluation and monitoring of patients with spinal muscular atrophy
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

GERMANY

Nordrhein-Westfalen
KÖLN

Spinal muscular atrophy: Molecular and functional analysis of a new SMA modifier
Institut für Humangenetik am Universitätsklinikum Köln
Institut für Humangenetik

ITALY

CAMPANIA
NAPOLI

Funded by an IRDiRC memberIdentification of new druggable targets and potential therapeutic compounds for Spinal Muscular Atrophy, using a C.elegans model of neurodegeneration
Istituto di Genetica e Biofisica "Adriano Buzzati Traverso" - CNR
Genetica Molecolare dei Nematodi

ITALY

FRIULI VENEZIA GIULIA
TRIESTE

Funded by an IRDiRC memberMolecular mechanism and therapeutic activity of modified U1 snRNAs in Spinal Muscular Athrophy
International Centre For Genetic Engineering And Biotechnology - ICGEB
Human Molecular Genetics Laboratory

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberRegulation of SMN2 splicing in cell and mouse models of spinal muscular atrophy
A.O.U. Policlinico di Tor Vergata - Facoltà di Medicina e Chirurgia
Laboratorio di Trasduzione del Segnale

ITALY

LAZIO
ROMA

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberDevelopment of an italian clinical network for spinal muscular atrophy
Policlinico Universitario "A. Gemelli"
U.O.C. Neuropsichiatria Infantile

ITALY

LOMBARDIA
MILANO

Genetic correction of human induced pluripotent stem cells from patients with proximal spinal muscular atrophy
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Dipartimento di Scienze Neurologiche

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberPeptide-conjugated morpholino for treatment of spinal muscular atrophy
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Dipartimento di Scienze Neurologiche

UNITED KINGDOM

Greater Manchester
ADDRESS: NOT PROVIDED - UK

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberMolecular mediators of the spinal muscular atrophy NMJ phenotype
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberAn orally bioavailable drug candidate for spinal muscular atrophy
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberTargeting a smn lncrna for the treatment of sma
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberLong-term effect of troponin activators for spinal muscular atrophy
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberSurvival motor neuron (smn) function in motoneuron development
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberSmn post-translational modification in spinal muscular atrophy
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberProtein-mediated membrane remodeling
Institution: Information not provided - US

FRANCE

ILE-DE-FRANCE
EVRY

GERMANY

Baden-Württemberg
FREIBURG

Natural History, Outcome measures in SMA type II and III
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Neuropädiatrie und Muskelerkrankungen

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberObservational longitudinal study of growth patterns, body composition, energy expenditure and dietary intake in Italian infants and children with spinal muscular atrophy type I and II
Università degli Studi di Milano - Scienze Biomolecolari e Biotecnologiche
Dipartimento di Scienze per gli Alimenti, la Nutrizione e l'Ambiente

FRANCE

ILE-DE-FRANCE
PARIS

Funded by an IRDiRC memberXenopus tropicalis model of spinal muscular atrophy with resipatory distress (SMARD1)
Université Paris Descartes UMR S-1124
Dégénérescence neuromusculaire et plasticité

FRANCE

OCCITANIE
MONTPELLIER

GERMANY

Bayern
MARTINSRIED

Cellular Toxicity of Polyglutamine Proteins in Neurodegenerative Disease
Max-Planck-Institut für Biochemie
Abteilung zelluläre Biochemie

GERMANY

Berlin
BERLIN

KCC K-Cl cotransporters and GABAergic transmission
Leibniz-Institut für Molekulare Pharmakologie
Department Physiology and Pathology of Ion Transport

ITALY

EMILIA ROMAGNA
ADDRESS: NOT PROVIDED - IT

ITALY

EMILIA ROMAGNA
MODENA

ITALY

LAZIO
ROMA

Funded by an IRDiRC memberMuscular RNA and transcriptome analysis as a tool for the identification of biomarkers in spinal muscular atrophy
Policlinico Umberto I
Dipartimento di Biotecnologie Cellulari ed Ematologia

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberClinical network and Registry for Trial Readiness in Spinal and Bulbar Muscle Atrophy
Fondazione IRCCS Istituto Neurologico "Carlo Besta"
U.O.C. Genetica Medica e Neurogenetica

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberMotoneuron degeneration in spinal and bulbar muscular atrophy: from the molecular mechanisms to the potential therapeutical approaches (terminated)
Università degli Studi di Milano - Scienze Farmacologiche e Biomolecolari
Centro di Eccellenza per le Malattie Neurodegenerative

ITALY

LOMBARDIA
MILANO

Funded by an IRDiRC memberMotor neuron degeneration in spinal and bulbar muscular atrophy: molecular approaches to counteract mutant androgen receptor neurotoxicity
Università degli Studi di Milano - Scienze Farmacologiche e Biomolecolari
Centro di Eccellenza per le Malattie Neurodegenerative

JAPAN

JAPAN
ADDRESS : NOT PROVIDED - JP

JAPAN

JAPAN
AICHI

Funded by an IRDiRC memberEstablishment of appropriate treatment for spinal and bulbar muscular atrophy
Nagoya University Graduate School of Medicine
Department of Neurology

JAPAN

JAPAN
AICHI

Funded by an IRDiRC memberInnovative therapy development for neuro-muscular degeneration in spinal and bulbar muscular atrophy
Nagoya University Graduate School of Medicine
Department of Neurology

UNITED KINGDOM

Greater London
LONDON

Funded by an IRDiRC memberThe Pathogenesis of Spinal and Bulbar Muscular Atrophy
University College London - UCL Darwin Building
University College London

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberNuclear mechanisms of polyglutamine toxicity in sbma
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberCellular and molecular studies of sbma neuromuscular disease
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberGeneration and characterization of a zebrafish model of sbma
Institution: Information not provided - US

UNITED STATES

Washington
ADDRESS: NOT PROVIDED - US

Funded by an IRDiRC memberRole of usp7 in pathogenicity of spinal and bulbar muscular atrophy
Institution: Information not provided - US

Multicentric Research projects