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A closer look at gene therapy with PTC Therapeutics (pharmaphorum media limited) View |
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Background on PTC and their Gene Therapy Program in Angelman Syndrome Dr Stephanie Ciarlone 2020 F (FAST) View |
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PTC Therapeutics (Translarna) [PPMD's 2015 Connect Conference] (Parent Project Muscular Dystrophy) View |
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In the Pipeline: Restoring Dystrophin - Sarepta Therapeutics (RNA Targeted Therapies) (Parent Project Muscular Dystrophy) View |
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Ataluren - the meta-analysis (Rare Disease Report) View |
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Genetic Engineering (Amoeba Sisters) View |
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Experimental Gene Therapy Effective in Giving Kids New Chance at Life (KPIX | CBS NEWS BAY AREA) View |
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Nestor Serrano u0026 Pat Furlong: Global Genes Tribute to Champions Gala (Global Genes - Allies in Rare Disease) View |
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PPMD 2018 Conference - Pat's Pearls (Parent Project Muscular Dystrophy) View |
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Trial Update: CAP-1002 - Capricor [PPMD's 2017 Connect Conference] (Parent Project Muscular Dystrophy) View |