Beta-sarcoglycanopathy is an under-researched and underfunded "orphan" disease. Our volunteer organization named Family Group of Beta-sarcoglycanopathy Onlus (GFB Onlus www.lgmd2e.org) was founded in 2013 with the aim of stimulating the scientific research on this disease. Since 2012 the families of the GFB ONLUS have been funding a gene therapy project led by prof. J. Mendell in Columbus Ohio USA. In 2014 there was then the creation of the LGMD EuroNET during the second conference organized in Venice.
Beta-sarcoglycanopathy: what’s new? / B. Vola, R. Maggi, M. Cerletti, Y. Torrente. ((Intervento presentato al 15. convegno Congresso Nazionale Associazione Italiana di Miologia AIM tenutosi a Napoli nel 2015.
Beta-sarcoglycanopathy: what’s new?
R. Maggi;Y. Torrente
2015
Abstract
Beta-sarcoglycanopathy is an under-researched and underfunded "orphan" disease. Our volunteer organization named Family Group of Beta-sarcoglycanopathy Onlus (GFB Onlus www.lgmd2e.org) was founded in 2013 with the aim of stimulating the scientific research on this disease. Since 2012 the families of the GFB ONLUS have been funding a gene therapy project led by prof. J. Mendell in Columbus Ohio USA. In 2014 there was then the creation of the LGMD EuroNET during the second conference organized in Venice.
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