未登录 Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort Duong, Tina; Kishnani, Priya S.; Haack, Kristina An; Foster, Meredith C.; Gibson, James B.; Wilson, Catherine; Hahn, Si Houn; Hillman, Richard; Kronn, David; Leslie, Nancy D.; Pena, Loren D. M.; Sparks, Susan E.; Stockton, David W.; Tanpaiboon, Pranoot; Day, John W.; Consortium, Pompe Advance Study 分享 收藏
Clinical characteristics and genotypes in the ADVANCE baseline data set, a comprehensive cohort of US children and adolescents with Pompe disease Kishnani, Priya S.; Gibson, James B.; Gambello, Michael J.; Hillman, Richard; Stockton, David W.; Kronn, David; Leslie, Nancy D.; Pena, Loren D. M.; Tanpaiboon, Pranoot; Day, John W.; Wang, Raymond Y.; Goldstein, Jennifer L.; Haack, Kristina An; Sparks, Susan E.; Zhao, Yang; Hahn, Si Houn 分享 收藏
Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study Hahn, Si Houn; Kronn, David; Leslie, Nancy D.; Pena, Loren D. M.; Tanpaiboon, Pranoot; Gambello, Michael J.; Gibson, James B.; Hillman, Richard; Stockton, David W.; Day, John W.; Wang, Raymond Y.; Haack, Kristina An; Shafi, Raheel; Sparks, Susan; Zhao, Yang; Wilson, Catherine; Kishnani, Priya S. 分享 收藏
Clinical characteristics and genotypes in the ADVANCE baseline dataset, a comprehensive cohort of us children and youth with Pompe disease Kishnani, Priya S.; Day, John W.; Gambello, Michael; Gibson, James B.; Hillman, Richard; Kronn, David; Leslie, Nancy; Pena, Loren; Stockton, David W.; Tanpaiboon, Pranoot; Wang, Raymond; Haack, Kristina An; Sparks, Susan; Zhao, Yang; Hahn, Sihoun 分享 收藏
52-week efficacy and safety profile of alglucosidase alfa produced at 4000 liter scale in US patients with Pompe disease: ADVANCE, a phase 4 open-label prospective study Hahn, Sihoun; Pena, Loren; Day, John W.; Gambello, Michael; Gibson, James B.; Hillman, Richard; Kronn, David; Stockton, David; Leslie, Nancy; Tanpaiboon, Pranoot; Wang, Raymond; Haack, Kristina An; Sparks, Susan; Zhao, Yang; Kishnani, Priya S. 分享 收藏
Newborn screening for Fabry disease: Is the A143T allele a pathogenic mutation or a pseudodeficiency allele? Atherton, Andrea M.; Doheny, Dana; Peck, Dawn; Christensen, Katherine; Smith, Kayla; Manwaring, Linda; Kiesling, Jami; Hillman, Richard; Font-Montgomery, Esperanza; Shinawi, Marwan; Grange, Dorothy K.; Desnick, Robert J.; Heese, Bryce A. 分享 收藏
The first two years of full population pilot newborn screening for lysosomal disorders: The Missouri experience Atherton, Andrea M.; Peck, Dawn; Christensen, Katherine; Smith, Kayla; Manwaring, Linda; Hopkins, Patrick; Rogers, Sharmini; Kiesling, Jami; Font-Montgomery, Esperanza; Hillman, Richard; Braddock, Stephen; Shinawi, Marwan; Grange, Dorothy K.; Heese, Bryce A. 分享 收藏
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