Recombinant human acid alpha-glucosidase - Major clinical benefits in infantile-onset Pompe disease

PS Kishnani; D Corzo; M Nicolino; B Byrne; H Mandel; WL Hwu; N Leslie; J Levine; C Spencer; M McDonald; J Li; J Dumontier; M Halberthal; YH Chien; R Hopkin; S Vijayaraghavan; D Gruskin; D Bartholomew; Ans van der Ploeg; JP Clancy; R Parini; G Morin; M Beck; GS De la Gastine; M Jokic; B Thurberg; S Richards; D Bali; M Davison; MA Worden; YT Chen; JE Wraith

doi:10.1212/01.wnl.0000251268.41188.04

Recombinant human acid alpha-glucosidase - Major clinical benefits in infantile-onset Pompe disease

PS Kishnani
, D Corzo
, M Nicolino
, B Byrne
, H Mandel
, WL Hwu
, N Leslie
, J Levine
, C Spencer
, M McDonald
, J Li
, J Dumontier
, M Halberthal
, YH Chien
, R Hopkin
, S Vijayaraghavan
, D Gruskin
, D Bartholomew
, Ans van der Ploeg
, JP Clancy

R Parini, G Morin, M Beck, GS De la Gastine, M Jokic, B Thurberg, S Richards, D Bali, M Davison, MA Worden, YT Chen, JE Wraith

Pediatrics

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

740 Citations (Scopus)

Original language	Undefined/Unknown
Pages (from-to)	99-109
Number of pages	11
Journal	Neurology
Volume	68
Issue number	2
DOIs	https://doi.org/10.1212/01.wnl.0000251268.41188.04
Publication status	Published - 2007

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

Research programs

EMC MM-01-54-01

Access to Document

10.1212/01.wnl.0000251268.41188.04

http://hdl.handle.net/1765/35672

Cite this

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., ... Wraith, JE. (2007). Recombinant human acid alpha-glucosidase - Major clinical benefits in infantile-onset Pompe disease. Neurology, 68(2), 99-109. https://doi.org/10.1212/01.wnl.0000251268.41188.04

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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 2007, 'Recombinant human acid alpha-glucosidase - Major clinical benefits in infantile-onset Pompe disease', Neurology, vol. 68, no. 2, pp. 99-109. https://doi.org/10.1212/01.wnl.0000251268.41188.04

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T1 - Recombinant human acid alpha-glucosidase - Major clinical benefits in infantile-onset Pompe disease

AU - Kishnani, PS

AU - Corzo, D

AU - Nicolino, M

AU - Byrne, B

AU - Mandel, H

AU - Hwu, WL

AU - Leslie, N

AU - Levine, J

AU - Spencer, C

AU - McDonald, M

AU - Li, J

AU - Dumontier, J

AU - Halberthal, M

AU - Chien, YH

AU - Hopkin, R

AU - Vijayaraghavan, S

AU - Gruskin, D

AU - Bartholomew, D

AU - van der Ploeg, Ans

AU - Clancy, JP

AU - Parini, R

AU - Morin, G

AU - Beck, M

AU - De la Gastine, GS

AU - Jokic, M

AU - Thurberg, B

AU - Richards, S

AU - Bali, D

AU - Davison, M

AU - Worden, MA

AU - Chen, YT

AU - Wraith, JE

PY - 2007

Y1 - 2007

U2 - 10.1212/01.wnl.0000251268.41188.04

DO - 10.1212/01.wnl.0000251268.41188.04

M3 - Article

C2 - 17151339

SN - 0028-3878

VL - 68

SP - 99

EP - 109

JO - Neurology

JF - Neurology

IS - 2

ER -