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7. Clinical trials In BMT: ensuring that rare diseases and rarer therapies are well done. Shurin S; Krischer J; Groft SC Biol Blood Marrow Transplant; 2012 Jan; 18(1 Suppl):S8-11. PubMed ID: 22226117 [No Abstract] [Full Text] [Related]
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19. Patient-reported outcomes in clinical trials of rare diseases. Basch E; Bennett AV J Gen Intern Med; 2014 Aug; 29 Suppl 3(Suppl 3):S801-3. PubMed ID: 25029974 [TBL] [Abstract][Full Text] [Related]
20. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. O'Reilly M; Kohn DB; Bartlett J; Benson J; Brooks PJ; Byrne BJ; Camozzi C; Cornetta K; Crystal RG; Fong Y; Gargiulo L; Gopal-Srivastava R; High KA; Jacobson SG; Jambou RC; Montgomery M; Rosenthal E; Samulski RJ; Skarlatos SI; Sorrentino B; Wilson JM; Xie Y; Corrigan-Curay J Hum Gene Ther; 2013 Apr; 24(4):355-62. PubMed ID: 23517518 [TBL] [Abstract][Full Text] [Related] [Next] [New Search]