Recombinant human acid {alpha}-glucosidase: Major clinical benefits in infantile-onset Pompe disease.

P S Kishnani; D Corzo; M Nicolino; B Byrne; H Mandel; W L Hwu; N Leslie; J Levine; C Spencer; M McDonald; J Li; J Dumontier; M Halberthal; Y H Chien; R Hopkin; S Vijayaraghavan; D Gruskin; D Bartholomew; A van der Ploeg; J P Clancy; R Parini; G Morin; M Beck; G S De la Gastine; M Jokic; B Thurberg; S Richards; D Bali; M Davison; M A Worden; Y T Chen; J E Wraith

doi:10.1212/01.wnl.0000407271.54424.a2

Recombinant human acid {alpha}-glucosidase: Major clinical benefits in infantile-onset Pompe disease.

P S Kishnani, D Corzo, M Nicolino, B Byrne, H Mandel, W L Hwu, N Leslie, J Levine, C Spencer, M McDonald, J Li, J Dumontier, M Halberthal, Y H Chien, R Hopkin, S Vijayaraghavan, D Gruskin, D Bartholomew, A van der Ploeg, J P ClancyR Parini, G Morin, M Beck, G S De la Gastine, M Jokic, B Thurberg, S Richards, D Bali, M Davison, M A Worden, Y T Chen, J E Wraith

Research output: Contribution to journal › Article › peer-review

Original language	English
Journal	Neurology
Volume	77
Issue number	17
DOIs	https://doi.org/10.1212/01.wnl.0000407271.54424.a2
Publication status	Published - 25 Oct 2011

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10.1212/01.wnl.0000407271.54424.a2

Improved clinical management of lysosomal disorders
Wraith, J. E. (Participant), Fairbairn, L. J. (Participant), (Participant), Bigger, B. (Participant) & (Participant)
Impact: Health impacts

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Kishnani, P. S., Corzo, D., Nicolino, M., Byrne, B., Mandel, H., Hwu, W. L., Leslie, N., Levine, J., Spencer, C., McDonald, M., Li, J., Dumontier, J., Halberthal, M., Chien, Y. H., Hopkin, R., Vijayaraghavan, S., Gruskin, D., Bartholomew, D., van der Ploeg, A., ... Wraith, J. E. (2011). Recombinant human acid {alpha}-glucosidase: Major clinical benefits in infantile-onset Pompe disease. Neurology, 77(17). https://doi.org/10.1212/01.wnl.0000407271.54424.a2

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title = "Recombinant human acid {alpha}-glucosidase: Major clinical benefits in infantile-onset Pompe disease.",

author = "Kishnani, {P S} and D Corzo and M Nicolino and B Byrne and H Mandel and Hwu, {W L} and N Leslie and J Levine and C Spencer and M McDonald and J Li and J Dumontier and M Halberthal and Chien, {Y H} and R Hopkin and S Vijayaraghavan and D Gruskin and D Bartholomew and {van der Ploeg}, A and Clancy, {J P} and R Parini and G Morin and M Beck and {De la Gastine}, {G S} and M Jokic and B Thurberg and S Richards and D Bali and M Davison and Worden, {M A} and Chen, {Y T} and Wraith, {J E}",

year = "2011",

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Kishnani, PS, Corzo, D, Nicolino, M, Byrne, B, Mandel, H, Hwu, WL, Leslie, N, Levine, J, Spencer, C, McDonald, M, Li, J, Dumontier, J, Halberthal, M, Chien, YH, Hopkin, R, Vijayaraghavan, S, Gruskin, D, Bartholomew, D, van der Ploeg, A, Clancy, JP, Parini, R, Morin, G, Beck, M, De la Gastine, GS, Jokic, M, Thurberg, B, Richards, S, Bali, D, Davison, M, Worden, MA, Chen, YT & Wraith, JE 2011, 'Recombinant human acid {alpha}-glucosidase: Major clinical benefits in infantile-onset Pompe disease.', Neurology, vol. 77, no. 17. https://doi.org/10.1212/01.wnl.0000407271.54424.a2

TY - JOUR

T1 - Recombinant human acid {alpha}-glucosidase: Major clinical benefits in infantile-onset Pompe disease.

AU - Kishnani, P S

AU - Corzo, D

AU - Nicolino, M

AU - Byrne, B

AU - Mandel, H

AU - Hwu, W L

AU - Leslie, N

AU - Levine, J

AU - Spencer, C

AU - McDonald, M

AU - Li, J

AU - Dumontier, J

AU - Halberthal, M

AU - Chien, Y H

AU - Hopkin, R

AU - Vijayaraghavan, S

AU - Gruskin, D

AU - Bartholomew, D

AU - van der Ploeg, A

AU - Clancy, J P

AU - Parini, R

AU - Morin, G

AU - Beck, M

AU - De la Gastine, G S

AU - Jokic, M

AU - Thurberg, B

AU - Richards, S

AU - Bali, D

AU - Davison, M

AU - Worden, M A

AU - Chen, Y T

AU - Wraith, J E

PY - 2011/10/25

Y1 - 2011/10/25

U2 - 10.1212/01.wnl.0000407271.54424.a2

DO - 10.1212/01.wnl.0000407271.54424.a2

M3 - Article

C2 - 22025457

SN - 1526-632X

VL - 77

JO - Neurology

JF - Neurology

IS - 17

ER -

Recombinant human acid {alpha}-glucosidase: Major clinical benefits in infantile-onset Pompe disease.

Access to Document

Impacts

Improved clinical management of lysosomal disorders

Cite this