未登录 Safety and Efficacy of DTX401, an AAV8-Mediated Liver-Directed Gene Therapy, in Adults With Glycogen Storage Disease Type I a (GSDIa) DTX401的安全性和有效性评估，该疗法为AAV8介导的肝脏靶向基因治疗，用于治疗成人1型a型糖原贮积症（GSDIa）。 Weinstein, David A.; Derks, Terry G.; Rodriguez-Buritica, David F.; Ahmad, Ayesha; Couce, Maria-Luz; Mitchell, John J.; Riba-Wolman, Rebecca; Mount, Malaya; Sallago, Julieta Bonvin; Ross, Katalin M.; van Der Klauw, Melanie M.; de Boer, Foekje; van Der Schaaf, Caroline; Saavedra, Heather; Martinez-Olmos, Miguel; Atanga, Elvis; Hosseini, Asad; Mitragotri, Deepali; Crombez, Eric 分享 收藏
Long-term safety and efficacy of DTX301 in adults with late-onset ornithine transcarbamylase (OTC) deficiency: A Phase 1/2 trial Thomas, Janet; Tan, Wen-Hann; Khan, Aneal; Konczal, Laura; Breilyn, Margo Sheck; Hualde, Leticia Ceberio; Couce, Marie Luz; Geberhiwot, Tarekegn; Guffon, Nathalie; Harding, Cary; Patra, Kaushik; Merritt, J. Lawrence, II; Crombez, Eric 分享 收藏
Long-term velaglucerase alfa treatment in children with Gaucher disease type 1 naive to enzyme replacement therapy or previously treated with imiglucerase Smith, Laurie; Rhead, William; Charrow, Joel; Shankar, Suma P.; Bavdekar, Ashish; Longo, Nicola; Mardach, Rebecca; Harmatz, Paul; Hangartner, Thomas; Lee, Hak-Myung; Crombez, Eric; Pastores, Gregory M. 分享 收藏
Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease Elstein, Deborah; Mehta, Atul; Hughes, Derralynn A.; Giraldo, Pilar; Charrow, Joel; Smith, Laurie; Shankar, Suma P.; Hangartner, Thomas N.; Kunes, Yune; Wang, Nan; Crombez, Eric; Zimran, Ari 分享 收藏
Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials Hughes, Derralynn A.; Gonzalez, Derlis E.; Lukina, Elena A.; Mehta, Atul; Kabra, Madhulika; Elstein, Deborah; Kisinovsky, Isaac; Giraldo, Pilar; Bavdekar, Ashish; Hangartner, Thomas N.; Wang, Nan; Crombez, Eric; Zimran, Ari 分享 收藏
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Velaglucerase alfa enzyme replacement therapy compared with imiglucerase in patients with Gaucher disease Ben Turkia, Hadhami; Gonzalez, Derlis E.; Barton, Norman W.; Zimran, Ari; Kabra, Madhulika; Lukina, Elena A.; Giraldo, Pilar; Kisinovsky, Isaac; Bavdekar, Ashish; Ben Dridi, Marie-Francoise; Gupta, Neerja; Kishnani, Priya S.; Sureshkumar, E. K.; Wang, Nan; Crombez, Eric; Bhirangi, Kiran; Mehta, Atul 分享 收藏
Enzyme replacement therapy with velaglucerase alfa in Gaucher disease: Results from a randomized, double-blind, multinational, Phase 3 study Gonzalez, Derlis E.; Ben Turkia, Hadhami; Lukina, Elena A.; Kisinovsky, Isaac; Ben Dridi, Marie-Francoise; Elstein, Deborah; Zahrieh, David; Crombez, Eric; Bhirangi, Kiran; Barton, Norman W.; Zimran, Ari 分享 收藏
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Safety and efficacy of velaglucerase alfa in Gaucher disease type 1 patients previously treated with imiglucerase Zimran, Ari; Pastores, Gregory M.; Tylki-Szymanska, Anna; Hughes, Derralynn A.; Elstein, Deborah; Mardach, Rebecca; Eng, Christine; Smith, Laurie; Heisel-Kurth, Margaret; Charrow, Joel; Harmatz, Paul; Fernhoff, Paul; Rhead, William; Longo, Nicola; Giraldo, Pilar; Ruiz, Juan A.; Zahrieh, David; Crombez, Eric; Grabowski, Gregory A. 分享 收藏
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Two-Year Efficacy And Safety Of Velaglucerase Alfa In Patients With Type 1 Gaucher Disease Switching From Imiglucerase: Phase III Trial HGT-GCB-039 And Extension Crombez, Eric; Kishnani, Priya; Ben Turkia, Hadhami; Gonzalez, Derlis; Zimran, Ari; Kabra, Madhulika; Lukina, Elena; Giraldo, Pilar; Kisinovsky, Isaac; Bavdekar, Ashish; Ben Dridi, Marie-Francoise; Gupta, Neerja 分享 收藏
Enzyme replacement therapy with velaglucerase alfa significantly improves key clinical parameters in type 1 Gaucher disease: Positive results from a randomized, double-blind, global, phase III study Zimran, Ari; Gonzalez, Derlis; Lukina, Elena A.; Ben Dridi, Marie-Franoise; Kisinovsky, Isaac; Crombez, Eric; Bhirangi, Kiran 分享 收藏
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