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GENETICS IN MEDICINE, ISSN 1098-3600, 11/2017, Volume 19, Issue 11, pp. 1281 - 1282
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2018, Volume 123, Issue 2, pp. 85 - 91
Journal Article
MOLECULAR GENETICS AND METABOLISM, ISSN 1096-7192, 11/2017, Volume 122, Issue 3, pp. 99 - 107
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2016, Volume 117, Issue 2, pp. 66 - 83
Journal Article
Journal Article
Molecular Genetics and Metabolism Reports, ISSN 2214-4269, 09/2019, Volume 20, p. 100475
Enzyme replacement therapy (ERT) with rhGAA has improved clinical outcomes in infantile Pompe disease (IPD). A subset of CRIM-positive IPD patients develop... 
Pompe disease | Anti-rhGAA Ig antibodies | Neuromuscular disease | Glycogen storage disease type II | Antidrug antibodies | Enzyme replacement therapy | ALGLUCOSIDASE ALPHA | GLYCOGEN | RECOMBINANT | GENETICS & HEREDITY | ACID ALPHA-GLUCOSIDASE | CLINICAL-OUTCOMES
Journal Article
by Porth, O and Chatterjee, K and Narayan, R and Gammie, CF and Mizuno, Y and Anninos, P and Baker, JG and Bugli, M and Chan, CK and Davelaar, J and Del Zanna, L and Etienne, ZB and Fragile, PC and Kelly, BJ and Liska, M and Markoff, S and McKinney, JC and Mishra, B and Noble, SC and Olivares, H and Prather, B and Rezzolla, L and Ryan, BR and Stone, JM and Tomei, N and White, CJ and Younsi, Z and Akiyama, K and Alberdi, A and Alef, W and Asada, K and Azulay, R and Baczko, AK and Ball, D and Balokovic, M and Barrett, J and Bintley, D and Blackburn, L and Boland, W and Bouman, KL and Bower, GC and Bremer, M and Brinkerink, CD and Brissenden, R and Britzen, S and Broderick, AE and Broguiere, D and Bronzwaer, T and Byun, DY and Carlstrom, JE and Chael, A and Chatterjee, S and Chen, MT and Chen, YJ and Cho, I and Christian, P and Conway, JE and Cordes, JM and Crew, GB and Cui, YZ and De Laurentis, M and Deane, R and Dempsey, J and Desvignes, G and Doeleman, SS and Eatough, RP and Falcke, H and Fish, VL and Fomalont, E and Fraga-Encinas, R and Freeman, B and Friberg, P and Fromm, CM and Gomez, JL and Galison, P and Garcia, R and Gentaz, O and Georgiev, B and Goddi, C and Gold, R and Gu, MF and Gurwell, M and Hada, K and Hecht, MH and Hesper, R and Ho, LC and Ho, P and Honma, M and Huang, CWL and Huang, L and Hughes, DH and Ikeda, S and Inoue, M and Issaoun, S and James, DJ and Jannuzi, BT and Janssen, M and Jeter, B and Jiang, W and Johnson, MD and ... and Event Horizon Telescope Collaborat and The Event Horizon Telescope Collaboration and Lawrence Livermore National Lab. (LLNL), Livermore, CA (United States)
ASTROPHYSICAL JOURNAL SUPPLEMENT SERIES, ISSN 0067-0049, 08/2019, Volume 243, Issue 2, p. 26
Journal Article
JCI INSIGHT, ISSN 2379-3708, 07/2016, Volume 1, Issue 11
BACKGROUND. Enzyme replacement therapy (ERT) has prolonged survival and improved clinical outcomes in patients with infantile Pompe disease (IPD), a rapidly... 
ANTIBODIES | MEDICINE, RESEARCH & EXPERIMENTAL | ALGLUCOSIDASE ALPHA | INFANTS | ACID ALPHA-GLUCOSIDASE | BORTEZOMIB | ENZYME REPLACEMENT THERAPY | CLINICAL-OUTCOMES | CHILDREN
Journal Article
NPJ genomic medicine, ISSN 2056-7944, 2019, Volume 4, Issue 1, pp. 7 - 7
The response of childhood acute lymphoblastic leukemia (ALL) to dexamethasone predicts the long-term remission outcome. To explore the mechanisms of... 
Mutation | Kinases | Leukemia | Cloning
Journal Article