2 European Projects Found
Searched on 125080 European Projects
FINISHED
Clinical trial of gene therapy for MPS VI - a severe lysosomal storage disorder (MEUSIX)
Start date: Dec 1, 2012, End date: Nov 30, 2017,
"Mucopolysaccharidosis VI (MPS VI, or Maroteaux-Lamy syndrome; OMIM #253200) is a rare lysosomal storage disease caused by deficient activity of arylsulfatase B (ARSB). MPS VI is characterized by growth retardation, corneal clouding, cardiac valve disease, organomegaly, skeletal dysplasia, without central nervous system involvement. Thus, systemic therapies targeting peripheral organs have the pot ...
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A European Consortium for Lysosomal Disorders (EUCLYD)
Start date: May 1, 2008, End date: Apr 30, 2011,
"The EUCLYD consortium aims at developing a scientific network among outstanding communities of basic and clinical investigators in five European countries (Italy, Netherlands, UK, Sweden and Germany) to study various aspects of lysosomal storage diseases (LSD). LSDs are rare disorders (overall prevalence of 1/5000 to 1/8000), each due to a specific lysosomal enzyme deficiency, leading to intracel ...
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