DUCHENNE MUSCULAR DYSTROPHY Experts

• Research area/topic:
  Cardiac calcium channel abnormalities in Duchenne Muscular Dystrophy
• Medizinische Universität Wien
  
• karlhein********@***uniwien.ac.at

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• Research area/topic:
  Targeted delivery of antisense-mediated exon skipping therapy in transgenic pig model of DMD
• University of Alberta
  

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• Research area/topic:
  An autologous cell therapy approach to treat Duchenne muscular dystrophy using engineered muscle tissue derived from induced pluripotent stem cells.
• Centre hospitalier universitaire Sainte-Justine
  

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• Research area/topic:
  Strategies for therapy of respiratory muscle failure in muscular dystrophy
• McGill University, Meakins-Christie Laboratories
  
• Basil.******@***ill.ca

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• Research area/topic:
  Genetic Regulation of Myogenesis
• Ottawa Hospital Research Institute
  
• mrud*****@***i.ca

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• Research area/topic:
  Post-transcriptional Regulation of Utrophin in Skeletal Muscle: Implications for New Therapeutic Strategies for Duchenne Muscular Dystrophy
• University of Ottawa
  
• jas***@***tawa.ca

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• Research area/topic:
  Can laminin-111 be used to treat Duchenne Muscular Dystrophy alone or in combination with myoblast transplantation?
• Centre de Recherche du Centre Hospitalier Universitaire de Québec, CHUQ - (CHUL) Centre hospitalier de l'Université Laval
  Centre Hospitalier Universitaire de Québec, CHUQ - (CHUL) Centre hospitalier de l'Université Laval
  
• jacques_p*********@***hul.ulaval.ca

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• Research area/topic:
  Growth arrest and osteoporosis in Duchenne muscular dystrophy patients treated with glucocorticoids
• Mount Sinai Hospital
  
• gry****@***enfeld.ca

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• Research area/topic:
  Rescue_ribosome : Rescue Pathways for unrecycled ribosomes - FR
• I2BC - UMR 9198, Institut de Biologie Intégrative de la Cellule (I2BC)
  

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• Research area/topic:
  TOLGEN: In vivo AAV-based gene transfer to the muscle: Deciphering host immune responses - FR
• Université de Nantes
  
• Oumeya*******@***v_nantes.fr

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• Research area/topic:
  Pre-clinical and clinical development of AAV-mediated microdystrophin delivery for gene therapy of DMD
• Université de Nantes
  
• moul****@***.edu

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• Research area/topic:
  Dystrophins In The Nervous System: From Neurophysiology to Molecular Therapy
• UMR 9197, CNRS Université Paris-Sud, Université Paris-Sud
  
• cyrille.********@***sud.fr

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• Research area/topic:
  Implementation of non-invasive prenatal diagnosis of rare and severe monogenic diseases
• CHU Paris Centre - Hôpital Cochin, Site Cochin
  
• juliette********@***p.fr

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• Research area/topic:
  ENDOSTEM: Activation of vasculature associated stem cells and muscle stem cells for the repair and maintenance of muscle tissue
• Université Pierre et Marie Curie - Paris 6
  

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• Research area/topic:
  SIRD: Stimulating Intrinsic Repair for DMD - FR
• Institut Cochin (INSERM U 1016 - CNRS UMR 8104 - Paris Descartes UMR-S1016)
  
• fabien.********@***erm.f

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• Research area/topic:
  TAMDMD : Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial -DE-
• DRK Kliniken Berlin Westend
  
• a.m****@***_kliniken_westend.de

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• Research area/topic:
  Progression study on the characterization of skeletal muscles by magnetic resonance imaging in Duchenne muscular dystrophy
• ZSEER - Zentrum für Seltene Erkrankungen Erlangen
  
• regina.t********@***erlangen.de

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• Research area/topic:
  PRO-DMD-01: A prospective natural history study of progression of physical impairment, activity limitation and quality of life in Duchenne Muscular Dystrophy
• Universitätsklinikum Freiburg
  Zentrum für Kinder- und Jugendmedizin Freiburg
  Zentrum für Kinder- und Jugendmedizin Freiburg
  Zentrum für Kinder- und Jugendmedizin Freiburg
  
• janbernd.*********@***klinik_freiburg.de

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• Research area/topic:
  BIO-NMD: identifying and validating pre-clinical biomarkers for diagnostics and therapeutics of neuromuscular disorders
• Dipartimento di Riproduzione e Accrescimento, A.O.U. di Ferrara - Polo Chimico-Bio-Medico
  
• f**@***fe.it

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• Research area/topic:
  A novel in vitro Duchenne Muscular Dystrophy cardiomyopathy model: human iPSC-derived cardiomyocytes for mechanistic studies
• Università degli studi di Firenze, Università degli studi di Firenze-Dip. di Medicina Sperimentale e Clinica
  

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• Research area/topic:
  USEFUL: Sistema assistivo centrato sull'utente per il supporto delle funzioni del braccio in soggetti affetti da patologie neuromoscolari
• Politecnico di Milano
  
• alessandra**********@***imi.it

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• Research area/topic:
  OptiStem: Optimisation of stem cell therapy for clinical trials of degenerative skin and muscle diseases (TERMINATED)
• Divisione di Medicina Rigenerativa, IRCCS Ospedale San Raffaele
  
• cossu.******@***.it

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• Research area/topic:
  Evaluation of bone turnover, bone metabolism, bone density, and fractures in children with Duchenne Muscular Dystrophy and possible side effects of long-term corticosteroid therapy (BON-DMD)
• Dipartimento di Scienze Mediche e Riabilitative ad indirizzo Endocrino-Metabolico, IRCCS Istituto Auxologico Italiano - Ospedale San Michele
  
• ml.bi*****@***ologico.it

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• Research area/topic:
  BIO-NMD: biomarker discovery and validation on animal models (WP5)
• Università degli Studi di Padova- Polo A.Vallisneri
  
• paolo.*******@***pd.it

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• Research area/topic:
  Pre-clinical evaluation of biocompatible nanoparticles as delivery system of 20-methyl-phosphorothioate antisense oligoribonucleotides for exon skippin- mediated dystrophin in restoration
• Università degli Studi di Padova- Polo A.Vallisneri
  
• brag****@***.unipd.it

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• Research area/topic:
  MYORES: Multi-organismic approach to study normal and aberrant muscle development, function and repair
• Università degli Studi di Padova- Polo A.Vallisneri
  

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• Research area/topic:
  Assessment of upper limb function in non-ambulatory patients with Duchenne Muscular Dystrophy
• Dipartimento di Neuroscienze e Neuroriabilitazione, IRCCS Ospedale Pediatrico Bambino Gesù - SEDE SAN PAOLO
  IRCCS Ospedale Pediatrico Bambino Gesù - SEDE GIANICOLO
  
• enricosilv**********@***g.net

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• Research area/topic:
  Long term natural history in Duchenne muscular dystrophy
• Policlinico Universitario "A. Gemelli"
  
• eugeniomar**********@***catt.it

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• Research area/topic:
  RNA-based studies of Duchenne Muscular Dystrophy: post-transcriptional control and role of non coding RNAs in normal and dystrophic muscle development
• Sapienza Università di Roma
  
• irene.*******@***roma1.it

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• Research area/topic:
  Role of HDAC4 in muscular dystrophy and regeneration
• Sapienza Università di Roma - Sezione di Ricerca "Valdoni Lab"
  
• viviana*******@***roma1.it

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• Research area/topic:
  Assessment of upper limb function in non ambulant Duchenne muscular dystrophy
• Università Cattolica del Sacro Cuore
  
• marik******@***unicatt.it

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• Research area/topic:
  Cell therapy for muscle dystrophies: delivery of myogenic precursor cells and amniotic stem cells via polymeric scaffolds
• Azienda ULSS 9 - Ospedale di Treviso
  
• pdec****@***mail.com

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• Research area/topic:
  BIO-NMD: exploratory biomarker validation in humans (WP4)
• LUMC - Leids Universitair Medisch Centrum
  

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• Research area/topic:
  Antisense therapy for several major rare diseases
• LUMC - Leids Universitair Medisch Centrum
  

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• Research area/topic:
  Do women from families with a Duchenne patient know that they can be a carrier and are possibly at risk for having a son with Duchenne musclular dystrophy?
• Afdeling Klinische Genetica, LUMC - Leids Universitair Medisch Centrum
  

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• Research area/topic:
  Do women from families with a Duchenne patient know that they can be a carrier and are possibly at risk for having a son with Duchenne musclular dystrophy?
• Afdeling Klinische Genetica, LUMC - Leids Universitair Medisch Centrum
  LUMC - Leids Universitair Medisch Centrum
  LUMC - Leids Universitair Medisch Centrum
  
• M.H.Br******@***c.nl

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• Research area/topic:
  Development of an exoskeleton for the arms for boys with Duchenne Muscular Dystrophy
• Radboudumc - Oost
  
• Imelda.*******@***boudumc.nl

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• Research area/topic:
  QUADRES: QUAntification of Dystrophin Restoration by Exon Skippping
• Instituto de Investigación sanitaria BioCruces
  
• virginia.are*************@***kidetza.eus

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• Research area/topic:
  Regenerative potential of the obestatin/GPR39 system on the treatment of pathologies characterized by muscle atrophy
• IDIS - Instituto de Investigación Sanitaria de Santiago de Compostela
  
• jesus******@***.es

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• Research area/topic:
  TAMDMD : Tamoxifen in Duchenne muscular dystrophy - a randomised placebo controlled phase 2 trial
• University of Basel Children's Hospital
  Universitäts-Kinderspital beider Basel UKBB
  
• dirk.f******@***b.ch

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• Research area/topic:
  Characterisation of skeletal development and the use of anabolic agents in murine models of Duchenne muscular dystrophy
• University of Edinburgh
  

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• Research area/topic:
  Enhancing the Therapeutic Functionality of Adeno-associated Virus (AAV) Vectors Encoding Dystrophin for Duchenne Muscular Dystrophy Gene Therapy
• Royal Holloway, University of London
  

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• Research area/topic:
  Secondary Osteoporosis and Its Therapy in Duchenne Muscular Dystrophy (ScOT-DMD)
• University of Glasgow, Royal Hospital For Children, NHS Greater Glasgow & Clyde
  RHC & QEUH Campus, Greater Glasgow & Clyde NHS Health Board, Queen Elizabeth University Hospital Campus
  RHC & QEUH Campus, Greater Glasgow & Clyde NHS Health Board, Queen Elizabeth University Hospital Campus
  South Glasgow University Hospital, Royal Hospital For Children, NHS Greater Glasgow & Clyde
  University of Glasgow, Kelvin Building
  
• faisal******@***sgow.ac.uk

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• Research area/topic:
  Advances in oligonucleotide-mediated exonskipping for DMD and related disorders - WP3 - Natural history extension
• UCL Institute of Child Health, University College London
  Department of Paediatrics, Hammersmith Hospital
  

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• Research area/topic:
  Neuromuscular Rare Disease Translational Research in patients with Duchenne Muscular Dystrophy
• University College London Hospitals, NHS Foundation Trust
  
• RDGove******@***h.nhs.uk

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• Research area/topic:
  BIOIMAGE-Neuromuscular Diseases
• Newcastle University, Newcastle upon Tyne Hospitals NHS Trust
  
• olav.ve********@***.ac.uk

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• Research area/topic:
  MICA: The role of utrophin in DMD and its therapeutic potential
• Department of Physiology Anatomy & Genetics, Le Gros Clark Building - University of Oxford
  University of Oxford
  
• kay.d*****@***g.ox.ac.uk

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• Research area/topic:
  Antisense nucleic acid splice correction therapy for Duchenne muscular dystrophy and related disorders
• Department of Physiology, Anatomy and Genetics, Le Gros Clark Building - University of Oxford
  University of Oxford
  
• matthe******@***g.ox.ac.uk

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• Research area/topic:
  Regulation of dystroglycan function and its implications in Duchenne Muscular Distrophy
• Firth Court, University of Sheffield
  
• s.wi****@***ffield.ac.uk

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