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Výzkumné projekty
KANADA
Ontario
TORONTO
Inherited bone marrow failure syndromes: from genomic discoveries to biology
Hospital for Sick Children, Research Institute
RAKOUSKO
STEIERMARK
GRAZ
CRISPR/Cas9 Genom-Engineering zur Erforschung von MPNs (myeloproliferativen Neoplasien)
Medizinische Universität Graz
Klinische Abteilung für Hämatologie
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
Besremi-PASS: A Prospective, Multicentre, Non-interventional, Observational, Post-authorisation Safety Study of Ropeginterferon alfa-2b in Polycythaemia Vera Patients - AT
Institution: Information not provided - AT
FRANCIE
ILE-DE-FRANCE
FONTENAY-AUX-ROSES CEDEX
Identification par cribles fonctionnels à haut débit de gènes modificateurs de la maladie de Fanconi
DRF/Institut François-Jacob CEA Paris-Saclay
Laboratoire d'Exploration Fonctionnelle des Génomes - LEFG
FRANCIE
NOUVELLE AQUITAINE
BORDEAUX
Rôle des sous-populations de monocytes dans la thrombose associée aux néoplasmes myéloprolifératifs (MonSThr)
CHU de Bordeaux-GH Pellegrin
Service d'hématologie biologique
NĚMECKO
Berlin
ADDRESS: NOT PROVIDED - DE
JAKoMo: Eine prospektive, zweiarmige, nicht-interventionelle Studie zu JAKAVI® (Ruxolitinib) bei Patienten mit Myelofibrose
Institution: Information not provided - DE
NĚMECKO
Sachsen-Anhalt
HALLE (SAALE)
Phäno- und Genotypen von Patienten mit JAK2-Mutation mit oder ohne thromboembolischem Ereignis - Ein Projekt der Ostdeutschen Studiengruppe Hämatologie und Onkologie (OSHO)
Universitätsklinikum Halle (Saale)
Krukenberg-Krebszentrum Halle (KKH)
ITÁLIE
EMILIA ROMAGNA
MELDOLA
Integrazione multiomica e caratterizzazione multilivello dei disordini e delle neoplasie maligne ematologiche
IRST - Istituto Scientifico Romagnolo per lo studio e la cura dei Tumori
IRCCS Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori
ITÁLIE
LOMBARDIA
MILANO
RBDD: Rede Database delle Malattie Rare della Coagulazione (coordination)
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Laboratorio di Patologie Molecolari Applicate alla Clinica
ŠVÝCARSKO
Suisse Alémanique
BASEL
Next generation advanced therapies for fight Beta-hemoglobinopathies via rational intervention in beta-globin regulatory network
Departement Biosysteme (D-BSSE)
Departement für Biosysteme und Ingenieurwissenschaften ETH Zürich
FRANCIE
ILE-DE-FRANCE
PARIS
Maladies hématologiques constitutionnelles: recherche clinique, thérapeutique; biologie moléculaire
GHU AP-HP Nord. Université de Paris - Hôpital Saint-Louis
Service d'hématologie pédiatrique
PORTUGALSKO
SUL
LISBOA
Investigação Molecular em Hemoglobinopatias
Instituto Nacional de Saúde Dr. Ricardo Jorge - Lisboa
Departamento de Genética
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
Adakveo (crizanlizumab) PRegnancy outcomes Intensive Monitoring (PRIM) - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
WIEN
RIBOEUROPE: The European Ribosomopathy Consortium - AT
AKH BT 25.3
Ludwig Boltzmann Institute for Rare and Undiagnosed Diseases
RAKOUSKO
WIEN
WIEN
Identifizierung von genetischen Suppressoren von Diamond-Blackfan-Anämie
Vienna Biocenter
IMBA - Institute of Molecular Biotechnology
BELGIE
HAINAUT
GOSSELIES
RIBOEUROPE: Le Consortium Européen de la Ribosomopathie - BE
Biopark campus
The 'RNA Metabolism' Lab - Université Libre de Bruxelles (ULB)
BELGIE
HAINAUT
GOSSELIES
DBAGenCure: Thérapie génique à médiation lentivirale pour l'anémie de Blackfan-Diamond : études précliniques d'innocuité et d'efficacité - BE
Biopark campus
The 'RNA Metabolism' Lab - Université Libre de Bruxelles (ULB)
KANADA
Ontario
TORONTO
Pathobiology and robosomal insufficiency in Shwachman-Diamond syndrome
Hospital for Sick Children, Research Institute
FINSKO
Finland
HELSINKI
Genomic editing for treating hemoglobin diseases
University of Helsinki
Stem Cells and Metabolism Research Program
FRANCIE
GRAND-EST
STRASBOURG
TC NER: Transcription stress Counteracted by Nutritional interventions of Exceptional importance for rare DNA Repair diseases - FR
CHU de Strasbourg - Hôpital de Hautepierre
Service de Neuropédiatrie
FRANCIE
ILE-DE-FRANCE
LE KREMLIN-BICÊTRE
Dépistage et prise en charge clinique des maladies de l'hémostase acquises liées à des auto-anticorps
APHP. Université Paris-Saclay, Hôpital Bicêtre
Service d'Hématologie
FRANCIE
ILE-DE-FRANCE
PARIS
Physiopathologie de la métaplasie myéloïde avec myélofibrose
AP-HP.Sorbonne Université - Hôpital Saint-Antoine
UF de cytogénétique onco-hématologique
FRANCIE
ILE-DE-FRANCE
PARIS
Diagnostic prénatal non invasif de la drépanocytose par séquençage nouvelle génération (NGS)
CHU Paris - Hôpital Robert Debré
UF de Génétique Moléculaire
FRANCIE
ILE-DE-FRANCE
PARIS
Microcephaly, Fanconi Anemia and Praxial Disorders (MicroFancII)
CHU Paris - Hôpital Robert Debré
UF de Génétique clinique
FRANCIE
ILE-DE-FRANCE
PARIS
DBAGenCure: Lentiviral-mediated gene therapy for Diamond Blackfan Anemia: Preclinical Safety and Efficacy Studies - FR
CHU Paris - Hôpital Robert Debré
Service d'Hématologie biologique
FRANCIE
ILE-DE-FRANCE
PARIS
RIBOEUROPE: The European Ribosomopathy Consortium - FR
Faculté de Médecine Paris Diderot Paris 7 - site Bichat
Centre de Recherche sur l'Inflammation (UMR 1149 Inserm - Université Paris Diderot - ERL CNRS 8252)
FRANCIE
ILE-DE-FRANCE
PARIS
FANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - FR
GHU AP-HP Nord. Université de Paris - Hôpital Saint-Louis
Laboratoire d'hématologie
FRANCIE
ILE-DE-FRANCE
PARIS
CRISPR/Cas9 mediated induction of fetal hemoglobin synthesis for the treatment of beta-hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163
FRANCIE
ILE-DE-FRANCE
PARIS
Development of Innovative Therapeutic Strategies for Beta-Hemoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163
FRANCIE
ILE-DE-FRANCE
PARIS
Amélioration des stratégies de thérapie génique basées sur l'utilisation des cellules souches hématopoïétiques pour le traitement des ß-hémoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163
FRANCIE
ILE-DE-FRANCE
PARIS
Développement d'une approche universelle de « prime editing » des bêta-hémoglobinopathies
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163
FRANCIE
ILE-DE-FRANCE
PARIS
Recherche biologique sur le syndrome d'Evans (ES) et l'anémie hémolytique autoimmune (AIHA) chez l'enfant
Institut National de la Transfusion Sanguine (INTS)
Physiologie du globule rouge normal et pathologique
FRANCIE
ILE-DE-FRANCE
VILLEJUIF
Physiopathologie de la métaplasie myéloïde avec myélofibrose
GHU Paris-Sud - Hôpital Paul Brousse
Les cellules souches: de leurs niches à leurs applications thérapeutiques
FRANCIE
OCCITANIE
TOULOUSE
RIBODBA: anémie de Diamond Blackfan: mécanismes moléculaires d'une maladie ribosomique
Université Paul Sabatier
Laboratoire de Biologie Moléculaire Eucaryote - UMR 5099
FRANCIE
OCCITANIE
TOULOUSE
DEVDBA: Ontogeny as a critical determinant of DBA sensitivity in red blood cells - FR
Université Toulouse III
Unité de biologie Moléculaire, cellulaire et du développement
NĚMECKO
Baden-Württemberg
FREIBURG
Eine prospektive, internationale, multizentrische Verlaufbeobachtungsstudie bei Patienten mit kombiniertem T-Zell Immundefekt (profound combined immunodeficiency : P-CID)
CCI am Universitätsklinikum Freiburg
Centrum für Chronische Immundefizienz
NĚMECKO
Baden-Württemberg
FREIBURG
DBAGenCure: Lentiviral-vermittelte Gentherapie für Diamond Blackfan Anemia: Präklinische Sicherheits- und Wirksamkeitsstudien
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Pädiatrische Hämatologie und Onkologie
NĚMECKO
Baden-Württemberg
FREIBURG
RIBOEUROPE: Das Europäische Ribosomopathie-Konsortium
Zentrum für Kinder- und Jugendmedizin Freiburg
Klinik für Pädiatrische Hämatologie und Onkologie
NĚMECKO
Baden-Württemberg
HEIDELBERG
ADDRess: Translationale Forschung für Personen mit DNA Reparaturdefekten
Deutsches Krebsforschungszentrum
KITZ - Hopp-Kindertumorzentrum Heidelberg
NĚMECKO
Baden-Württemberg
HEIDELBERG
DBAGenCure: Lentiviral-vermittelte Gentherapie für Diamond Blackfan Anemia: Präklinische Sicherheits- und Wirksamkeitsstudien
GeneWerk GmbH
NĚMECKO
Baden-Württemberg
HEIDELBERG
ADDRess: Translationale Forschung für Personen mit DNA Reparaturdefekten
Universitäts-Frauenklinik Heidelberg
Frauenheilkunde und Geburtshilfe
NĚMECKO
Bayern
WÜRZBURG
ADDRess: Translationale Forschung für Personen mit DNA Reparaturdefekten
Universität Würzburg - Biozentrum
Institut für Humangenetik
NĚMECKO
Hessen
LANGEN
Präklinische Gentherapie von Fanconi-Anämie mit transposon-basierten Ansätzen
Paul-Ehrlich-Institut
Abteilung Medizinische Biotechnologie
NĚMECKO
Mecklenburg-Vorpommern
GREIFSWALD
Mutationsspektrum und klinische Symptomatik bei Patienten mit genetisch bedingten Hämostasestörungen
Institut für Humangenetik der Universitätsmedizin Greifswald
Institut für Humangenetik
NĚMECKO
Nordrhein-Westfalen
DÜSSELDORF
ADDRess: Translationale Forschung für Personen mit DNA Reparaturdefekten
Universitätsklinikum Düsseldorf
Institut für Pathologie
ITÁLIE
LAZIO
ROMA
Progressi nella diagnosi della talassemia e delle emoblobinopatie: sviluppo di tecniche innovative attraverso l'uso combinato di termogravimetria e chemiometria
ISS - Istituto Superiore di Sanità
Centro Nazionale per la Ricerca e la Valutazione preclinica e clinica dei Farmaci
ITÁLIE
LAZIO
ROMA
RegistRare: the new National Platform dedicated to Rare Diseases Registries
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare
ITÁLIE
LAZIO
ROMA
Migliorare gli standard di qualità dei laboratori italiani che svolgono test genetici per le malattie rare
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare
ITÁLIE
LOMBARDIA
MILANO
TC NER: Transcription stress Counteracted by Nutritional interventions of Exceptional importance for rare DNA Repair diseases - IT
IFOM - Istituto FIRC di Oncologia Molecolare
Firc Institute of Molecular Oncology (IFOM)
ITÁLIE
LOMBARDIA
PAVIA
Un modello di midollo osseo basato sulla seta per predire la risposta individuale a farmaci vecchi e nuovi per aumentare la conta piastrinica nelle piastrinopenie ereditarie
Fondazione IRCCS Policlinico San Matteo
Clinica Medica III
KOREJSKÁ REPUBLIKA
KOREA, REPUBLIC OF
SEOUL
Utveckling av cell och genterapi för blodsjukdomar, i synnerhet Gauchers och Blackfan-Diamond sjukdomar
Seoul National University Hospital
Molecular Diagnostics Laboratory
PORTUGALSKO
SUL
LISBOA
DESENVOLVIMENTO E VALIDAÇÃO DE PREDITORES PRECOCES DA VASO-OCLUSÃO NUM MODELO MENDELIANO DE DOENÇA VASCULAR
Instituto Nacional de Saúde Dr. Ricardo Jorge - Lisboa
Departamento de Genética
RUMUNSKO
BUCURESTI
BUCURESTI
Identificarea si caracterizarea moleculara a mutatiilor care codifica lantul beta - globinei la pacientii cu talasemie din romania
Fundeni Clinical Institute
Department of Hematology
ŠPANĚLSKO
Andalucía
MÁLAGA
Medicina de sistemas aplicada a la identificación de nuevas dianas terapéuticas en enfermedades raras de base genética
Universidad de Málaga. Facultad de Ciencias
Departamento de Biología Molecular y Bioquímica
ŠPANĚLSKO
Cataluña
BARCELONA
Medicina personalizada en pacientes con enfermedad de células falciformes basada en el diagnóstico integrado de metodologías de nueva generación
Vall d'Hebron Institut de Recerca VHIR
Vall d'Hebron Institut de Recerca
ŠPANĚLSKO
Madrid
MADRID
DBAGenCure: Lentiviral-vermittelte Gentherapie für Diamond Blackfan Anemia: Präklinische Sicherheits- und Wirksamkeitsstudien
CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
Terapias innovadoras en el sistema hematopoyético
ŠPANĚLSKO
Madrid
MADRID
Efectos del agonista no peptídico del receptor de trombopoyetina (Eltrombopag) en la hematopoyesis de los pacientes con anemia de Fanconi
Hospital Infantil Universitario Niño Jesús
Servicio de Hematología y Hemoterapia
ŠPANĚLSKO
Madrid
MADRID
FANEDIT: Edición génica, una nueva estrategia terapéutica para la anemia de Fanconi - ES
Hospital Infantil Universitario Niño Jesús
Servicio de Hematología y Hemoterapia
ŠPANĚLSKO
Madrid
MADRID
DBAGenCure: Terapia génica mediada por lentivirus para la anemia de Blackfan Diamond: Estudios preclínicos de seguridad y eficacia - ES
Hospital Materno Infantil Gregorio Marañón
Servicio de Oncología y Hematología Pediátricas y del Adolescente
ŠVÉDSKO
Region Skåne
LUND
DEVDBA: Ontogeny as a critical determinant of DBA sensitivity in red blood cells - SE
Lund University
Department of Clinical Sciences
ŠVÉDSKO
Region Skåne
LUND
Bakteriella virulensfaktorer, medfödd immunitet och protrombotiska mekanismer vid njursjukdomar
Lund University
Department of Clinical Sciences - Division of Pediatrics
ŠVÝCARSKO
Suisse Alémanique
BERN
Thrombotic thrombocytopenic purpura - role of ADAMTS13 and long-term outcome
Universitätsspital Inselspital
Department of Hematology and Central Hematology Laboratory
ŠVÝCARSKO
Suisse Alémanique
ZURICH
FANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia - CH
ETH Zurich - Hönggerberg campus
Institute of Molecular Health Sciences
SPOJENÉ KRÁLOVSTVÍ
Greater London
LONDON
The genetic basis and pathophysiology of dyskeratosis congenita, aplastic anaemia, myelodysplasia and related disorders. Study of the genes DKC1, TERC, TERT, TIN2, NOP10, NHP2, C16orf57 and TCAB1 and new gene discovery
Barts and The London School of Medicine and Dentistry
Centre for Paediatrics
SPOJENÉ STÁTY
North Carolina
CHAPEL HILL
Prospective Study in Sickle Cell Anemia: Determine Biomarkers of Endothelial Function Changes in Chronic Kidney Disease
University of North Carolina
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Therapeutics for rare and neglected diseases - science
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Preclinical innovation
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Phenotypic assay design and development for rare and neglected diseases
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Repurposing an eu therapeutic for hemoglobinopathies
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Fanconi anemia:genotype-phenotype correlations
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Genetic epidemiology of complex traits
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Hematopoietic stem cell biology
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Insights into sickle cell trait and sickle cell disease
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Red cell biology
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Understanding the relationships between race; ethnicity; ancestry and genomics
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
The impact of childhood chronic disease on child patient reported outcomes
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Mri-based quantitative brain oxygen metabolism identifying high risk of infarct recurrence in sickle cell anemia
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Age-dependence of cerebral oxygen metabolism and stroke risk in pediatric sickle cell disease
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Fa ddr pathway in germline integrity
Institution: Information not provided - US
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
Argatroban Monitoring in Critically Ill Patients: Evaluation of a Novel Ecarin-based Bedside Test
Institution: Information not provided - AT
RAKOUSKO
WIEN
WIEN
Prospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie
FRANCIE
ILE-DE-FRANCE
LE KREMLIN-BICÊTRE
Caractérisation immunologique et régulation de la réponse immunitaire anti-facteur VIII dans les hémophilies A, et anti-facteur IX dans les hémophilies B
APHP. Université Paris-Saclay, Hôpital Bicêtre
Service d'Hématologie
FRANCIE
ILE-DE-FRANCE
PARIS
Mise en place du diagnostic prénatal non invasif des maladies monogéniques rares et sévères
AP-HP.Centre - Université de Paris - Hôpital Cochin
Service de Médecine Génomique des Maladies de Système et d'Organe
FRANCIE
ILE-DE-FRANCE
PARIS
Cytopénies Auto-immunes: génétique et mécanismes physiopathologiques du syndrome d'Evans pédiatrique
IMAGINE - Institut des Maladies Génétiques
Institut IMAGINE - INSERM U1163
FRANCIE
NOUVELLE AQUITAINE
BORDEAUX
Safety of CRISPR-Cas9 nuclease use
Université de Bordeaux ISPED
Centre de recherche INSERM U1219
FRANCIE
PROVENCE-ALPES-COTE D'AZUR
NICE
Role de l'interaction Piezo1/KCNN4 dans la physiopathologie du globule rouge
Université de Nice Sophia-Antipolis - Faculté des sciences
iBV - Institut de Biologie Valrose
NĚMECKO
Baden-Württemberg
FREIBURG
GAIN: Deutsches Netzwerk für die Erforschung und Therapieoptimierung von Patienten mit Multi-Organ-Autoimmunerkrankungen
CCI am Universitätsklinikum Freiburg
Centrum für Chronische Immundefizienz
NĚMECKO
Baden-Württemberg
HEIDELBERG
NGS2025: Evaluation einer Erweiterung des Neugeborenenscreenings um 28 zusätzliche Zielkrankheiten
Zentrum für Kinder- und Jugendmedizin des Universitätsklinikums Heidelberg
Klinik für Kinderheilkunde I - Sektion für Neuropädiatrie und Stoffwechselmedizin
NĚMECKO
Baden-Württemberg
TÜBINGEN
AWISPECS: Die Rolle des erworbenen von-Willebrand-Syndroms bei kardiochirurgischen Eingriffen im Säuglingsalter
Universitätsklinik für Kinder- und Jugendmedizin Tübingen
Abteilung für Kinderkardiologie, Pulmologie, Intensivmedizin
NĚMECKO
Bayern
MÜNCHEN
Etablierung von Oberflächenplasmonenresonanz (SPR) Tests zur verbesserten Diagnostik von Autoimmunerkrankungen
Klinikum rechts der Isar der Technischen Universität München
Arbeitsgruppe Biosensorik
NĚMECKO
Niedersachsen
HANNOVER
GAIN: Deutsches Netzwerk für die Erforschung und Therapieoptimierung von Patienten mit Multi-Organ-Autoimmunerkrankungen
Medizinische Hochschule Hannover
Klinik für Pädiatrische Pneumologie, Allergologie und Neonatologie
NĚMECKO
Niedersachsen
HANNOVER
GAIN: Deutsches Netzwerk für die Erforschung und Therapieoptimierung von Patienten mit Multi-Organ-Autoimmunerkrankungen
Medizinische Hochschule Hannover
Klinik für Rheumatologie und Immunologie
NĚMECKO
Nordrhein-Westfalen
KÖLN
REACT-2020: Retrospektive Analyse der Wirksamkeit von Caplacizumab bei der Behandlung der erworbenen thrombotischen thrombozytopenischen Purpura (aTTP)
Universitätsklinikum Köln
Klinik II für Innere Medizin - Nephrologie, Rheumatologie, Diabetologie und allgemeine Innere Medizin
NĚMECKO
Schleswig-Holstein
KIEL
GAIN: Deutsches Netzwerk für die Erforschung und Therapieoptimierung von Patienten mit Multi-Organ-Autoimmunerkrankungen
Universitätsklinikum Schleswig-Holstein - Campus Kiel
Klinik für Innere Medizin I
IRSKO
County Dublin
DUBLIN
The Low Von Willebrand factor Ireland Cohort of Kids (LoVIC-K) study
Royal College of Surgeons of Ireland
Molecular and Cellular Theraputics
ITÁLIE
LIGURIA
GENOVA
Studio osservazionale della durata di 12 mesi finalizzato a definire la prevalenza ed i criteri diagnostici della Ipertensione Polmonare (PAH) nei pazienti affetti da Thalassemia Major ed intermedia
Ospedali Galliera
S.S.D. Ematologia
ITÁLIE
LOMBARDIA
MILANO
Valutazione della secrezione dell'ormone della crescita e riserva surrenale, in parallelo con paramentri cardiaci ed ossei in pazienti adulti con Talassemia
IRCCS Istituto Auxologico Italiano
Dipartimento di Endocrinologia e Malattie del Ricambio
ITÁLIE
LOMBARDIA
MILANO
Sviluppo di vettori lentivirali per il trasferimento genetico nel fegato ed applicazioni alla terapia dell' Emofilia
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica
NORSKO
Nord-Norge
TROMSØ
Undersøkelse av karbohydrater bundet til antistoff rettet mot blodplateantigen
UNN - Universitetssykehuset Nord-Norge
Diagnostisk klinikk
NORSKO
Nord-Norge
TROMSØ
Plater og placenta - samspill for livet
UNN - Universitetssykehuset Nord-Norge
Diagnostisk klinikk
NORSKO
Nord-Norge
TROMSØ
Cellulære responser ved blodplatemangel hos foster og nyfødt
UNN - Universitetssykehuset Nord-Norge
Diagnostisk klinikk
PORTUGALSKO
SUL
LISBOA
Hemocromatose hereditária não-clássica e outras doenças genéticas raras associadas a distúrbios na homeostase do ferro
Instituto Nacional de Saúde Dr. Ricardo Jorge - Lisboa
Departamento de Genética
ŠPANĚLSKO
Madrid
MADRID
Papel de la glicosilación plaquetaria en la función de las plaquetas y en la respuesta inmune en pacientes con trombocitopenia inmune
IdiPAZ - Instituto de Investigación Sanitaria del Hospital Universitario La Paz
Grupo de Coagulopatías y alteraciones de la hemostasia
ŠPANĚLSKO
Madrid
MADRID
Perfiles cuantitativos de las proteínas FH/FHRS del complemento como biomarcadores de predisposición y pronostico en el síndrome hemolítico-urémico primario y secundario
IdiPAZ - Instituto de Investigación Sanitaria del Hospital Universitario La Paz
Laboratorio de Inmunopatología del Complemento
ŠPANĚLSKO
Madrid
MADRID
Mejorando la capacidad diagnóstica y el conocimiento de la patogénesis del síndrome antifosfolipídico primario mediante proteómica y transcriptómica
Instituto de Investigación Hospital 12 de Octubre
Fundación para la Investigación Biomédica del Hospital Universitario 12 de Octubre
ŠVÝCARSKO
Suisse Alémanique
ZÜRICH
The effect of cofactors and vitamins on homocysteine and methylmalonic acid metabolism in health and disease
Universitäts - Kinderspital Zürich - Eleonorenstiftung
Abteilung für Stoffwechselkrankheiten
SPOJENÉ KRÁLOVSTVÍ
Greater London
LONDON
Liver Disease in Pregnancy
King's College London
King's College London Headquarters
SPOJENÉ KRÁLOVSTVÍ
Tyne & Wear
NEWCASTLE UPON TYNE
The Role of Membrane Attack Complex in Atypical Haemolytic Uraemic Syndrome
Institute of Cellular Medicine, Newcastle University, Medical School
Institute of Cellular Medicine
SPOJENÉ STÁTY
Connecticut
CHESHIRE
CCRN 1093 (aHUS): An observational, non-interventional multicenter, multinational study of patients with Atypical HemolyticUremic Syndrome (aHUS Registry)
Alexion Pharmaceuticals, Inc.
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
AHEAD: ADVATE Hemophilia A Outcome Database - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
HEM-POWR: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
Eine prospektive, nicht-interventionelle Studie zur Beobachtung der Wirksamkeit von AFSTYLA® bei Patienten mit Hämophilie A - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
Surveillance Of Emicizumab-Treated Patients: An Analysis of the EUHASS Pharmacovigilance Registry - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
HA-SAFE: Observational study evaluating long-term safety of real-world treatment with damoctocog alfa pegol in previously treated patients with hemophilia A - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
A post authorization safety surveillance registry in previously untreated patients with severe hemophilia A in usual care settings - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
Emicizumab Use in Pediatric Patients in the Real World: an Analysis of the PedNet Registry - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
Hemlibra Survey to Prescribers and Patients/Carers to Evaluate Awareness, Knowledge and Compliance to Additional Risk Minimization Measures - AT
Institution: Information not provided - AT
KANADA
Ontario
KINGSTON
Study of the factor VIII life cycle and its implications for factor VIII immunogenicity
Queen's University
Department of Pathology and Molecular Medicine
FRANCIE
AUVERGNE-RHONE-ALPES
ADDRESS: NOT PROVIDED - FR
HEM-POWR: Etude observationnelle évaluant l'efficacité et la sécurité d'un traitement dans le monde réel avec Damoctocog Alfa Pegol chez des patients atteints d'hémophilie A ayant déjà été traités - FR
Institution: Information not provided - FR
FRANCIE
GRAND-EST
ILLKIRCH-GRAFFENSTADEN
Recherche de la (ou des) mutation(s) génétique(s) responsable(s) du syndrome de Scott
Faculté de Pharmacie de Strasbourg
Laboratoire de Bioimagerie et Pathologies - LBP
FRANCIE
ILE-DE-FRANCE
CRÉTEIL
Facteurs prédictifs de la réponse au traitement et impact du Rituximab sur les sous-populations lymphocytaires B et T au cours du Purpura thrombopénique immunologique de l'adulte (Etude RITUX-PTI)
Hôpitaux Universitaires Henri Mondor
Service de Médecine interne
FRANCIE
NOUVELLE AQUITAINE
PESSAC
Thrombopénies acquises chroniques de signification indeterminée: physiopathologie et biomarqueur
CHU de Bordeaux-GH Sud - Hôpital Haut-Lévêque
Unité de Cardiopathies congénitales de l'enfant et de l'adolescent
NĚMECKO
Bayern
MÜNCHEN
GEPHARD: German Paediatric Haemophilia Research Database (GEPHARD) Studie: Neu diagnostizierte Hämophilie
Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital
Hämophiliezentrum München - Pädiatrische Hämostaseologie
NĚMECKO
Hessen
FRANKFURT AM MAIN
AHA_IIT: Langzeitbeobachtung von Therapie und Outcome der erworbenen Hämophilie A
Universitätsklinikum Frankfurt
Schwerpunkt Hämostaseologie/Hämophiliezentrum Frankfurt
NĚMECKO
Hessen
MÖRFELDEN-WALLDORF
MOTIVATE: MOdern Behandlung von Inhibitor-PositiVe-Patienten mit Hämophilie A - Eine internationale Beobachtungsstudie
Hämophilie-Zentrum Rhein Main GmbH
NĚMECKO
Nordrhein-Westfalen
BONN
Enzymologische und strukturbiologische Aufklärung der Funktion des Vitamin K-Zyklus
Universitätsklinikum Bonn (AöR)
Institut für Experimentelle Hämatologie und Transfusionsmedizin
ITÁLIE
UMBRIA
PERUGIA
Identificazione di sottotipi di cellule dendritiche come chiave per l'induzione di tolleranza al FVIII nell'emofilia A
Università degli Studi di Perugia
Dipartimento di Medicina Sperimentale
NORSKO
Østlandet
OSLO
Skandinavisk populasjonsbasert-PK studie hos pasienter med hemofili
Oslo Universitetssykehus HF, Rikshospitalet
Pediatrisk forskningsinstitutt
NORSKO
Østlandet
OSLO
Faktor VII-mangel (CRISPR): Forsøk på å reparere genskade hos pasienter med faktor VII-mangel
Oslo University Hospital, Ullevaal
Oslo University Hospital, Ullevål
ŠPANĚLSKO
Cataluña
BARCELONA
PEAK Registry: Registro longitudinal global de deficiencia de piruvato quinasa
Hospital Universitari Vall d'Hebron
Servicio de Hematología y Hemoterapia
ŠPANĚLSKO
Comunidad Valenciana
VALENCIA
Tratamiento profiláctico personalizado con Advate® en pacientes con hemofilia A grave o moderada
Hospital Universitario y Politécnico La Fe
Servicio de Farmacia, Área Clínica del Medicamento
ŠPANĚLSKO
Madrid
ALCORCÓN
Descifrando el papel de factor-H related proteins en las enfermedades renales relacionadas con el complemento
Hospital Universitario Fundación Alcorcón
Servicio de Nefrología
ŠPANĚLSKO
Madrid
MADRID
Caracterización farmacocinético de la población con hemofilia A en España usando una aplicación médica en línea basada en un modelo de población publicado y un algoritmo bayesiano
Hospital Universitario La Paz
Servicio de Hematología
ŠPANĚLSKO
Madrid
MADRID
Descifrando el papel de factor-H related proteins en las enfermedades renales relacionadas con el complemento
Instituto de Investigación Hospital 12 de Octubre
Grupo de investigación en Nefrología Clínica
ŠPANĚLSKO
Madrid
VILLAVICIOSA DE ODÓN
Eficacia de un tratamiento profiláctico utilizando el factor de coagulación de vida media prolongada (LME) para mejorar la salud musculoesquelética de pacientes adultos con hemofilia. Estudio multicéntrico observacional
Universidad Europea de Madrid. Facultad de Ciencias de la Salud
Departamento de Fisioterapia
ŠPANĚLSKO
Murcia
MURCIA
Caracterización functional-molecular de pacientes con TPC y evaluación de la patogenicidad de nueva variantes Desarrollo de nuevos vectores lentivirals para corregir la trombastenia de Glanzmann
Hospital General Universitario Morales Meseguer
Servicio de Hematología y Oncología Médica
SPOJENÉ KRÁLOVSTVÍ
Greater Manchester
ADDRESS: NOT PROVIDED - UK
ADVATE Hemophilia A Outcome Database (AHEAD) - UK
Institution: Information not provided - UK
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
A-SURE: 24-month Prospective, Multicentre, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products in the Prophylactic Treatment of Patients With Haemophilia A - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
DYNAMO: The Dynamic Interplay Between Bleeding Phenotype and Baseline Factor Level in Moderate and Mild Hemophilia A and B - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
pathfinder9: A Multinational, Prospective, Open Labelled, Non-controlled, Non-interventional Post-authorisation Study of Turoctocog Alfa Pegol (N8-GP) During Long-term Routine Prophylaxis and Treatment of Bleeding Episodes in Patients With Haemophilia A - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
ADDRESS: NOT PROVIDED - AT
ECLIPSE: Screening/Observational Protocol to Determine Patient Eligibility for Inclusion in AAV Gene Therapy Clinical Trials - AT
Institution: Information not provided - AT
RAKOUSKO
WIEN
WIEN
A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults With Hemophilia - AT
Allgemeines Krankenhaus der Stadt Wien
Klinische Abteilung für Hämatologie und Hämostaseologie
KANADA
Ontario
OTTAWA
Nouvelles approches thérapeutiques pour cibler la myopathie GNE
Children's Hospital of Eastern Ontario
Division of Neurology
KANADA
Ontario
TORONTO
Canadian Hemophilia dose Escalation Primary Prophylaxis (CHPS) Observational Extension Study
Hospital for Sick Children, Research Institute
FRANCIE
AUVERGNE-RHONE-ALPES
PRAGUE
Role on Nonmuscle Myosin II in membrane trafficking and organ function
Faculty of Medicine and University Hospital Motol
Department of Pediatrics
FRANCIE
GRAND-EST
STRASBOURG
Syndrome de Bernard-Soulier et maladies liées au MYH9: étude des anomalies plaquettaires et de la thrombopoïèse, correction par thérapie génique
Établissement Français du Sang - Grand Est
UR U311 - Biologie et pharmacologie de l'hémostase et de la thrombose
NĚMECKO
Sachsen-Anhalt
HALLE (SAALE)
ProDGNE: Neuartige Therapieansätze zur Behandlung der GNE-Myopathie
Institut für Physiologische Chemie der Medizinische Fakultät
Institut für Physiologische Chemie
IZRAEL
ISRAEL
ADDRESS: NOT PROVIDED - IL
Zebrafish as a model system for GNE Myopathy
Institution: Information not provided - IL
IZRAEL
ISRAEL
JERUSALEM
Cellular Models to explore GNE functions in muscle
The Hebrew University of Jerusalem
Faculty of Medicine
ITÁLIE
SARDEGNA
CAGLIARI
ProDGNE: Nuovi approcci terapeutici indirizzati alla miopatia GNE - IT
Università degli Studi di Cagliari
Dipartimento di Scienze della Vita e dell'Ambiente
ŠPANĚLSKO
Madrid
MADRID
Estudio de efecto procoagulante del emicizumab y de su combinación con concentrados de factor para el tratamiento de sangrados en pacientes con hemofilia A grave
IdiPAZ - Instituto de Investigación Sanitaria del Hospital Universitario La Paz
Grupo de Coagulopatías y alteraciones de la hemostasia
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Cell biology of metabolic disorders
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Functional and translational studies of runx1 and cbfb in hematopoiesis
Institution: Information not provided - US
FRANCIE
GRAND-EST
STRASBOURG
Caractérisation des mutations responsables de la maladie de Bernard-Soulier (gènes GP1BA, GP1BB et GP9)
Établissement Français du Sang - Grand Est
UR U311 - Biologie et pharmacologie de l'hémostase et de la thrombose
ITÁLIE
LOMBARDIA
MILANO
Produzione vettori per la terapia genica (Sindrome di Wiskott-Aldrich e Leucodistrofia Metacromatica)
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Istituto San Raffaele Telethon per la Terapia Genica
ITÁLIE
LOMBARDIA
MILANO
Sindrome di Wiskott-Aldrich: caratterizzazione dei difetti immunologici e studi preclinici di terapia genica
Istituto San Raffaele Telethon per la Terapia Genica - TIGET
Unità di Ricerca Clinica
ŠVÉDSKO
Region Stockholm
HUDDINGE
Studier av de underliggande orsakerna till ändrad reglering av immunförsvaret hos barn med primära immunsjukdomar (Wiskott-Aldrich syndrom och X-bunden svår kongenital neutropeni)
Karolinska Institutet - Huddinge
Division of Clinical Chemistry
SPOJENÉ STÁTY
Maryland
BETHESDA
Novel Treatment for Hermansky-Pudlak Syndrome Pulmonary Fibrosis
National Center for Advancing Translational Sciences (NCATS-NIH)
Therapeutic Development Branch
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Gene therapy approaches for primary immunodeficiencies
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Molecular and clinical studies of primary immunodeficiencies
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
National ophthalmic disease genotyping and phenotyping network - eyegene
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Clinical research of oral connective tissue program
Institution: Information not provided - US
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
Protein trafficking in the endosomal-lysosomal system
Institution: Information not provided - US
ITÁLIE
LAZIO
ROMA
Role of Dystrophin-associated protein complex (DPC) in intracellular signaling and trafficking pathways
ISS - Istituto Superiore di Sanità
Centro Nazionale Malattie Rare
SPOJENÉ KRÁLOVSTVÍ
Cambridgeshire
CAMBRIDGE
Understanding granule disorders of myeloid cells by unravelling the interactome and function of the Nbeal2 protein
University of Cambridge
Departmet of Haematology
Multicentrické výzkumné projekty
- Universitätsklinikum des Saarlandes
- Institut für Molekulare Zellbiologie
- Institution: Information not provided - ES
- Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
- Laboratorio di Patologie Molecolari Applicate alla Clinica
- The Cyprus Institute of Neurology and Genetics
- Department of molecular genetics A
- Groupe hospitalier AP-HP.6 - Hôpital Tenon
- Laboratoire d'Hémostase et de Thrombose
- Université Paul Sabatier
- Laboratoire de Biologie Moléculaire Eucaryote - UMR 5099
- Zentrum für Kinder- und Jugendmedizin Freiburg
- Klinik für Pädiatrische Hämatologie und Onkologie
- CECAD Research Center
- Exzellenzcluster CECAD in der Universität zu Köln
- University of Athens - Medical school
- Laboratory of Biology
- University of Athens - Medical school
- Laboratory of Biology
- Università degli Studi di Ferrara
- Laboratorio di Biologia Molecolare dell'Emostasi e Trombosi
- Istituto San Raffaele Telethon per la Terapia Genica - TIGET
- Istituto San Raffaele Telethon per la Terapia Genica
- Fondazione IRCCS Policlinico San Matteo
- Laboratorio di Epidemiologia Clinica
- Università degli Studi di Bari Aldo Moro
- Dipartimento Integrato di Medicina
- CIEMAT - Centro de Investigaciones Energéticas Medioambientales y Tecnológicas
- Terapias innovadoras en el sistema hematopoyético
- Hospital Universitario Fundación Jiménez Díaz
- Cliniques universitaires Saint-Luc - UCLouvain
- Laboratory of Nephrology
- Université de Liège
- Centre d'Immunologie de Liège
- CHU Paris Nord-Val de Seine - Hôpital Xavier Bichat-Claude Bernard
- Fer et synthèse d'hème : génétique, physiologie et pathologie
- Zentrum für Kinder- und Jugendmedizin des Universitätsklinikums Heidelberg
- Klinik für Kinderheilkunde I - Sektion Pädiatrische Nephrologie
- Adienne S.R.L.
- Istituto San Raffaele Telethon per la Terapia Genica - TIGET
- Istituto San Raffaele Telethon per la Terapia Genica
- Prophylix Pharma AS
- Birmingham Children's Hospital NHS Foundation Trust
- Department of Nephrology
- APHP. Université Paris-Saclay, Hôpital Bicêtre
- Hémostase et Dynamique Cellulaire Vasculaire - INSERM U 770
- Faculté de Sciences Pharmaceutiques et Biologiques
- Unité de Technologies Chimiques et Biologiques pour la Santé
- GHU Paris-Sud - Hôpital Paul Brousse
- Les cellules souches: de leurs niches à leurs applications thérapeutiques
- Vall d'Hebron Institut de Recerca VHIR
- Grupo de investigación en terapia celular y genética
- Boston Children's Hospital
- Department of Pediatrics
- Institution: Information not provided - US
- Fondazione IRCCS Policlinico San Matteo
- Clinica Medica III
- Faculdade de Ciências Médicas - Universidade Nova de Lisboa
- Departamento de Ciências da Vida
- Istituto San Raffaele Telethon per la Terapia Genica - TIGET
- Unità di Ricerca Clinica
NĚMECKO
Saarland
HOMBURG
CoMMiTMenT: Combined Molecular Microscopy for Therapy and Personalized Medication in Rare Anaemias Treatments
ŠPANĚLSKO
Madrid
ADDRESS: NOT PROVIDED - ES
E-Enerca: New E-Health Services For The European Reference Network On Rare Anaemias
ITÁLIE
LOMBARDIA
MILANO
RBDD: Rede Database delle Malattie Rare della Coagulazione
KYPR
Cyprus
NICOSIA
ITHANET: réseau euro-méditerranéen des centres de recherche conduisant des recherches moléculaire et clinique sur la thalassémie et autres formes d'hémoglobinopathies
FRANCIE
ILE-DE-FRANCE
PARIS
Réseau de recherche clinique sur la drépanocytose
FRANCIE
OCCITANIE
TOULOUSE
RIBOEUROPE: The European Ribosomopathy Consortium
NĚMECKO
Baden-Württemberg
FREIBURG
EWOG-SAA 2010: Genetische und immunologische Charakterisierung der Schweren Aplastischen Anämie (SAA) bei Kindern und Jugendlichen
NĚMECKO
Nordrhein-Westfalen
KÖLN
TC NER: Transcription stress Counteracted by Nutritional interventions of Exceptional importance for rare DNA Repair diseases
ŘECKO
ATTIKI
ATHENS
GETHERTHAL: improvements of vector techology and safety for the gene therapy of thalassemia
ŘECKO
ATTIKI
ATHENS
GETHERTHALPLUS: Novel Gene Therapy for Thalassemia: Pre-clinical Development and Assessment in Animal and Stem Cell Models
ITÁLIE
EMILIA ROMAGNA
FERRARA
THALAMOSS: THALAssaemia MOdular Stratification System for personalized therapy of beta-thalassemia
ITÁLIE
LOMBARDIA
MILANO
DEVDBA: Ontogeny as a critical determinant of DBA sensitivity in red blood cells
ITÁLIE
LOMBARDIA
PAVIA
EUMNET: European Myelofibrosis Network
ITÁLIE
PUGLIA
BARI
DEEP: Valutazione sull'uso pediatrico del deferiprone
ŠPANĚLSKO
Madrid
MADRID
DBAGenCure: Lentiviral-mediated gene therapy for Diamond Blackfan Anemia: Preclinical Safety and Efficacy Studies
ŠPANĚLSKO
Madrid
MADRID
FANEDIT: Gene editing as a novel therapeutic strategy in Fanconi anemia
BELGIE
ARRONDISSEMENT BRUSSELS-CAPITAL
BRUSSELS
EUNEFRON: the European network for the study of orphan nephropathies
BELGIE
LIEGE
LIEGE
EUROTHYMAIDE: understanding the mechanisms underlying the development of autoimmune diseases, by exploring the major biological functions of the thymus
FRANCIE
ILE-DE-FRANCE
PARIS
HMA-IRON: towards improved diagnosis and treatment of rare inherited microcytic hypochromic anemias related to iron metabolism
NĚMECKO
Baden-Württemberg
HEIDELBERG
EURENOMICS: European Consortium for High-Throughput Research in Rare Kidney Diseases (TERMINATED)
ITÁLIE
LOMBARDIA
CAPONAGO
PRATH: preclinical study of recombinant human anti-C5 for the treatment of atypical hemolytic uremic syndrome (TERMINATED)
ITÁLIE
LOMBARDIA
MILANO
PERSIST: Persisting transgenesis (TERMINATED)
NORSKO
Nord-Norge
TROMSØ
PROFNAIT: Development of a prophylactic treatment for the prevention of fetal/neonatal alloimmune thrombocytopenia (FNAIT)
SPOJENÉ KRÁLOVSTVÍ
West Midlands
BIRMINGHAM
European Paediatric Research for Hemolytic Uremic Syndrome (HUS)
FRANCIE
ILE-DE-FRANCE
LE KREMLIN-BICÊTRE
SMART-HaemoCare: Small Antibody Fragment as Alternative Tools in Haemophilia Care
FRANCIE
ILE-DE-FRANCE
PARIS
TRANSPOSMART: An innovating platform using transposon and S/MAR for von Willebrand disease gene therapy
FRANCIE
ILE-DE-FRANCE
VILLEJUIF
InnovaLiv: Innovative strategies to generate human hepatocytes for treatment of metabolic Liver diseases: Tools for personalized cell therapy
ŠPANĚLSKO
Cataluña
BARCELONA
HEMO-iPS: Use of patient-specific induced pluripotent stem cells to improve diagnosis and treatment of hemophilia A
SPOJENÉ STÁTY
Massachusetts
BOSTON
PEAK Registry: Pyruvate Kinase Deficiency Global Longitudinal Registry
SPOJENÉ STÁTY
Washington
ADDRESS: NOT PROVIDED - US
HEM-POWR: Observational Study Evaluating Effectiveness and Safety of Real-World Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A
ITÁLIE
LOMBARDIA
PAVIA
EUPLANE: EUropean PLAtelet NEtwork for studying physiopathology of two inherited thrombocytopenias, THC2 and MYH9-RD, characterized by genetic alterations of RUNX1-target genes
PORTUGALSKO
SUL
LISBOA
ProDGNE: Novel therapeutic approaches to target GNE Myopathy
ITÁLIE
LOMBARDIA
MILANO