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A selection of FP7 projects

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Project Acronym Project Title
AIPGENE Augmenting PBGD expression in the liver as a Novel Gene therapy for Acute Intermittent Porphyria
ALPHA-MAN Clinical development of Enzyme Replacement Therapy in alpha-Mannosidosis patients using recombinant human enzyme.
BIO-NMD Identifying and validating pre-clinical biomarkers for diagnostics and therapeutics of Neuromuscular Disorders
CUREHLH European initiative to improve knowledge, treatment and survival of haemophagocytic syndromes in children
E-RARE-2 ERA-Net on Rare Diseases
EUNEFRON European Network for the Study of Orphan Nephropathies
EUCLYD A European Consortium for Lysosomal Disorders
EURIPFNET European IPF Network: Natural course, Pathomechanisms and Novel Treatment Options in Idiopathic Pulmonary Fibrosis
EURODSD Investigation of the molecular pathogenesis and pathophysiology of Disorders of Sex Development (DSD)
EURADRENAL Pathophysiology and natural course of autoimmune adrenal failure in Europe
EUROTRAPS Natural course, pathophysiology, models for early diagnosis, prevention and innovative treatment of TNF Receptor Associated Periodic Syndrome TRAPS with application for all hereditary recurrent fevers
EUCILIA Pathophysiology of rare diseases due to ciliary dysfunction: nephronophthisis, Oral-facial-digital type 1 and Bardet-Biedl syndromes
EFACTS European Friedreich's Ataxia Consortium for Translational Studies
EURO-PADNET The Pathophysiology and Natural Course of Patients with Primary Antibody Deficiencies (PAD)
FIGHT-MG Myasthenias, a group of immune mediated neurological diseases: from etiology to therapy.
GENEGRAFT Phase I/II ex vivo gene therapy clinical trial for recessive dystrophic epidermolysis bullosa using skin equivalent grafts genetically corrected with a COL7A1-encoding SIN retroviral vector
IMPACTT Immunoglobulin IgY pseudomonas A clinical trial for cystic fibrosis treatment
LEUKOTREAT Therapeutic challenge in Leukodystrophies: Translational and ethical research towards clinical trials
MABSOT Development of OPN-305 as an orphan drug for the treatment of Delayed Graft Function post solid organ transplantation
MEFOPA European Project on Mendelian Forms of Parkinson's Disease
NIMBL Nuclease Immune Mediated Brain and Lupus-like conditions (NIMBL): natural history, pathophysiology, diagnostic and therapeutic modalities with application to other disorders of autoimmunity
PADDINGTON Pharmacodynamic Approaches to Demonstration of Disease-Modification in Huntington's Disease by SEN0014196
PEMPHIGUS Pemphigus - From autoimmunity to disease
PRATH Preclinical study of Recombinant human Anti-C5 for the Treatment of atypical HUS
RAREDISEASEPLATFORM A European Platform of Integrated Information Services for Researchers in the Field of Rare Diseases and Orphan Drugs Supporting Team and Project Building.
RDCVF Rod-derived Cone Viability Factor
TREATRUSH Fighting blindness of Usher syndrome: diagnosis, pathogenesis and retinal treatment (TreatRetUsher)

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