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Financiado por um membro do IRDiRC =

Projetos de investigação

ESPANHA

Cataluña
L'HOSPITALET DE LLOBREGAT

ALEMANHA

Nordrhein-Westfalen
BONN

SMArtCARE : Clinical evaluation and monitoring of patients with spinal muscular atrophy
Universitäts-Kinderklinik Bonn
Abteilung für Neuropädiatrie

ALEMANHA

Nordrhein-Westfalen
KÖLN

The power of protective modifier NCALD to develop an efficient combinatorial therapy for spinal muscular atrophy
Institut für Humangenetik am Universitätsklinikum Köln
Institut für Humangenetik

BELGICA

ANTWERPEN
ANTWERPEN

CANADA

Ontario
OTTAWA

CANADA

Ontario
OTTAWA

Financiado por um membro do IRDiRCThe molecular pathogenesis of spinal muscular atrophy
Ottawa Hospital Research Institute
Regenerative Medicine

CANADA

Ontario
OTTAWA, ONTARIO

ESPANHA

Cataluña
LLEIDA

Financiado por um membro do IRDiRCNew therapies for treatment of spinal muscular atrophy: calpain inhibitors and modified mesenchymal stem cells
Instituto de Investigación Biomédica de Lleida (IRBLLEIDA)
Unidad de señalización neuronal

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

Financiado por um membro do IRDiRCMolecular mediators of the spinal muscular atrophy NMJ phenotype
Institution: Information not provided - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

Financiado por um membro do IRDiRCAn orally bioavailable drug candidate for spinal muscular atrophy
Institution: Information not provided - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

Financiado por um membro do IRDiRCTargeting a smn lncrna for the treatment of sma
Institution: Information not provided - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

Financiado por um membro do IRDiRCSurvival motor neuron (smn) function in motoneuron development
Institution: Information not provided - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

Financiado por um membro do IRDiRCProtein-mediated membrane remodeling
Institution: Information not provided - US

FRANCA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCA

AUVERGNE-RHONE-ALPES
LYON

Financiado por um membro do IRDiRCThe extracellular matrix collagen XV as a new player of motor axon development and regeneration: a functional study using zebrafish
Institut de Génomique Fonctionnelle de Lyon
Matrix Biology and Pathology, Managemant

FRANCA

ILE-DE-FRANCE
PARIS

Financiado por um membro do IRDiRCDeciphering respective contributions of macrophages and microglia to motor neuron degeneration in Spinal Muscular Atrophy and Amyotrophic Lateral Sclerosis with microfluidic devices
Institut du Cerveau et de la Moelle épinière (ICM) - Hôpital Pitié-Salpêtrière
Equipe "Causes de la SLA et mécanismes de la dégénérescence motoneuronale"

FRANCA

OCCITANIE
MONTPELLIER

Financiado por um membro do IRDiRCGenome-wide profiling of nuclear-body associated regions: relation to cellular physiopathology of the spinal muscular atrophy disease
UMR5535 Institut de génétique moléculaire de Montpellier (IGMM)
Architecture Génomique et Contrôle Epigénétique

FRANCA

OCCITANIE
MONTPELLIER

Financiado por um membro do IRDiRCDefining new functions for the SMN complex from yeast to human
UMR5535 Institut de génétique moléculaire de Montpellier (IGMM)
Assemblage et Trafic de Ribonucléoprotéines

FRANCA

PROVENCE-ALPES-COTE D'AZUR
NICE

Financiado por um membro do IRDiRCIn vivo and in vitro direct lineage reprogramming of neuronal and somatic cells to corticospinal motor neurons
Université de Nice Sophia-Antipolis - Faculté des sciences
iBV - Institut de Biologie Valrose

FRANCA

PROVENCE-ALPES-COTE D'AZUR
VALBONNE

Financiado por um membro do IRDiRCDeveloping combinatorial therapies for the treatment of spinal muscular atrophy
IPMC - Institut de Pharmacologie Moléculaire et Cellulaire
Physiopathologie des canaux Na+ et de l'excitabilité neuronale

ITALIA

CAMPANIA
NAPOLI

Financiado por um membro do IRDiRCIdentification 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

ITALIA

LAZIO
ROMA

In vitro and in vivo study of salbutamol a potential therapeutic agent for Spinal Muscular Atrophy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
U.O.C. Genetica Medica

ITALIA

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

ESPANHA

Madrid
MADRID

FINLANDIA

Finland
HELSINKI

FRANCA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCA

AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR

FRANCA

AUVERGNE-RHONE-ALPES
BRON

FRANCA

ILE-DE-FRANCE
EVRY

ITALIA

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

ITALIA

VENETO
PADOVA

Financiado por um membro do IRDiRCThe role of Raptor/mTORC1 in adult skeletal muscle
Venetian Institute of Molecular Medicine
Dipartimento di Scienze Biomediche

REINO UNIDO

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

CANADA

Ontario
TORONTO

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

Financiado por um membro do IRDiRCCellular and molecular studies of sbma neuromuscular disease
Institution: Information not provided - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

Financiado por um membro do IRDiRCA powerful web-based discovery platform for rare disease genetics
Institution: Information not provided - US

ESTADOS UNIDOS

Washington
ADDRESS: NOT PROVIDED - US

FRANCA

ILE-DE-FRANCE
PARIS

Financiado por um membro do IRDiRCSTaHR : Stimulation of Targeted Homologous Recombination for Gene Therapy - FR
Muséum National d'Histoire Naturelle
Laboratoire Structure et Instabilité des Génomes - INSERM U1154 - CNRS 7196

FRANCA

PAYS DE LA LOIRE
NANTES

Financiado por um membro do IRDiRCFROGH : FRench Regional Origins in Genetics for Health - FR
Institut de Recherche en Santé - Université de Nantes
L'Institut du thorax - Inserm UMR 1087 / CNRS UMR 6291

ITALIA

LAZIO
ROMA

Financiado por um membro do IRDiRCDiverse rare diseases caused by functional dysregulation of Rho GTPase-related proteins
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare

ITALIA

LAZIO
ROMA

Financiado por um membro do IRDiRCMuscular 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

ITALIA

LOMBARDIA
MILANO

Financiado por um membro do IRDiRCMotoneuron 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

JAPAO

JAPAN
AICHI

Financiado por um membro do IRDiRCDrug discovery for spinal-bulbar muscular atrophy using disease specific iPSCs
Aichi Medical University School of Medicine
Department of Neurology

JAPAO

JAPAN
AICHI

Financiado por um membro do IRDiRCEstablishment of appropriate treatment for spinal and bulbar muscular atrophy
Nagoya University Graduate School of Medicine
Department of Neurology

JAPAO

JAPAN
AICHI

Financiado por um membro do IRDiRCInnovative therapy development for neuro-muscular degeneration in spinal and bulbar muscular atrophy
Nagoya University Graduate School of Medicine
Department of Neurology

JAPAO

JAPAN
TOKYO

Financiado por um membro do IRDiRCFormation of integrated omics analysis base of refractory diseases in children and perinatal period
National Center for Child Health and Development
Research Institute

REINO UNIDO

Greater London
LONDON

Financiado por um membro do IRDiRCThe Pathogenesis of Spinal and Bulbar Muscular Atrophy
University College London - UCL Darwin Building
University College London

Projetos de investigação multicêntricos