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Academic Journal
Human gene therapy. 27(1):43-59
Academic Journal
Clinical immunology (Orlando, Fla.). 158(2):140-147
Academic Journal
Corti, M; Elder, ME; Falk, DJ; Lawson, L; Smith, BK; Nayak, S; Conlon, TJ; Clement, N; Erger, K; Lavassani, E; Green, MM; Doerfler, PA; Herzog, RW; Byrne, BJ
Molecular therapy. Methods & clinical development. 1
Electronic Resource
Stockton, DW; Kishnani, P; van der Ploeg, Ans; Llerena, J, Jr; Boentert, M; Roberts, M; Byrne, BJ; Araujo, R; Maruti, S S; Thibault, N; Verhulst, K; Berger, KI
Stockton , DW , Kishnani , P , van der Ploeg , A , Llerena , J J , Boentert , M , Roberts , M , Byrne , BJ , Araujo , R , Maruti , S S , Thibault , N , Verhulst , K & Berger , KI 2020 , ' Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start ' , Journal of Neurology , vol. 267 , no. 10 , pp. 3038-3053 .
Academic Journal
Molecular therapy. Methods & clinical development. 3:15053
Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent
Academic Journal
Nayak, S; Doerfler, PA; Porvasnik, SL; Cloutier, DD; Khanna, R; Valenzano, KJ; Herzog, RW; Byrne, BJ
PloS one. 9(6):e98336
Electronic Resource
Tremblay, JP; Xiao, X; Aartsma-Rus, A; Barbas, C; Blau, HM; Bogdanove, AJ; Boycott, K; Braun, S; Breakefield, XO; Bueren, JA; Buschmann, M; Byrne, BJ; Calos, M; Cathomen, T; Chamberlain, J; Chuah, M; Cornetta, K; Davies, KE; Dickson, JG; Duchateau, P; Flotte, TR; Gaudet, D; Gersbach, CA; Gilbert, R; Glorioso, J; Herzog, RW; High, KA; Huang, W; Huard, J; Joung, JK; Liu, D; Lochmüller, H; Lustig, L; Martens, J; Massie, B; Mavilio, F; Mendell, JR; Nathwani, A; Ponder, K; Porteus, M; Puymirat, J; Samulski, J; Takeda, S; Thrasher, A; Vandendriessche, T; Wei, Y; Wilson, JM; Wilton, SD; Wolfe, JH; Gao, G
Academic Journal
Kishnani, PS; Steiner, RD; Bali, D; Berger, K; Byrne, BJ; Case, L; Crowley, JF; Downs, S; Howell, RR; Kravitz, RM; Mackey, J; Marsden, D; Martins, AM; Millington, DS; Nicolino, M; O'Grady, G; Patterson, MC; Rapoport, DM; Slonim, A; Spencer, CT; Tifft, CJ; Watson, MS; ACMG Work Grp Management Pompe Dis
Academic Journal
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