2 projets européens trouvés
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TERMINÉ
VISION-DMD - Phase 2 Clinical Trials of VBP15: An Innovative Steroid-like Intervention on Duchenne Muscular Dystrophy (VISION DMD)
Date du début: 1 janv. 2016, Date de fin: 31 déc. 2019,
VISION-DMD aims to advance clinical development of the orphan drug VBP15 as a new therapy to revolutionise care for all patients with Duchenne muscular dystrophy (DMD) by 2020, in line with IRDiRC goals. DMD is an incurable, rare muscle wasting disease; boys progressively weaken, lose ambulation and death occurs by early adulthood. Corticosteroids (CS) are widely recognised to increase muscle stre ...
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TERMINÉ
"Nuclease Immune Mediated Brain and Lupus-like conditions (NIMBL): natural history, pathophysiology, diagnostic and therapeutic modalities with application to other disorders of autoimmunity" (NIMBL)
Date du début: 1 juin 2010, Date de fin: 30 nov. 2013,
"Nuclease Immune Mediated Brain and Lupus-like (NIMBL) conditions, comprising Aicardi-Goutieres Syndrome, Retinal Vasculopathy with Cerebral Leukodystrophy and some cases of Systemic Lupus Erythematosus, are devastating genetic disorders resulting in greatly reduced quality of life, high mortality especially in children, and significant risks of recurrence within affected families. NIMBL condition ...
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