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10.15252/emmm.201607293 http://scihub22266oqcxt.onion/10.15252/emmm.201607293 C5331198!5331198 !28148554     free     free     free Warning: file_get_contents(https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pubmed&id=28148554 &cmd=llinks): Failed to open stream: HTTP request failed! HTTP/1.1 429 Too Many Requests in C:\Inetpub\vhosts\kidney.de\httpdocs\pget.php on line 215       EMBO+Mol+Med 2017 ; 9 (3 ): 289-292 Nephropedia Template TP {{tp|p=28148554 |t=2017. Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis |pdf=|usr=}}{{28148554 }} gab.com Text Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis JO: EMBO Mol Med http://www.kidney.de/pget.php?&tw=1&pmid=28148554 #FOAvip The recent approval of Strimvelis, the first ex vivo gene therapy to gain marketing authorization (Schimmer & Breazzano, 2016), has drawn attention to Fondazione Telethon, the Italian charity that played a pivotal role in this effort. Although it is not uncommon that advanced therapies, such as Strimvelis, are developed by partnerships between academia and industry, direct involvement of a charity in key steps of this process is still unusual. Illustrating the strategies and operational model adopted by Fondazione Telethon to achieve its mission of supporting excellent research aimed at curing rare genetic diseases may elucidate some of the enabling factors behind the Strimvelis success story. Twit Text FOAVip Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis ????EMBO Mol Med http://www.kidney.de/pget.php?&tw=1&pmid=28148554 #FOAvip Twit Text # Free Paper on # # # # # LINK http://www.kidney.de/pget.php?&tw=1&pmid=28148554 #FOAvip English Wikipedia <ref>{{Cite pmid|28148554 }}</ref> Patient-driven search for rare disease therapies: the Fondazione Telethon success story and the strategy leading to Strimvelis #MMPMID28148554 Monaco L ; Faccio L EMBO Mol Med 2017[Mar]; 9 (3 ): 289-292 PMID28148554 show ga The recent approval of Strimvelis, the first ex vivo gene therapy to gain marketing authorization (Schimmer & Breazzano, 2016), has drawn attention to Fondazione Telethon, the Italian charity that played a pivotal role in this effort. Although it is not uncommon that advanced therapies, such as Strimvelis, are developed by partnerships between academia and industry, direct involvement of a charity in key steps of this process is still unusual. Illustrating the strategies and operational model adopted by Fondazione Telethon to achieve its mission of supporting excellent research aimed at curing rare genetic diseases may elucidate some of the enabling factors behind the Strimvelis success story. |*Rare Diseases [MESH]Patient-driven search for rare disease therapies: the Fondazione Telet(epmc).pdf DeepDyve Pubget Overpricing