G.P.11.06 Pharmacological chaperone therapy for Pompe disease: Phase 1 clinical trials and ex vivo response study results

Pine, C.W.; Nafar, Hadis; Palling, David J.; Slonim, Alfred E.; Pestronk, Alan; Byrne, Barry; Kishnani, Priya S.; Ranes, Brian; Kissling, Christian; Schwab, Robert S.; Connolly, S.; Sitaraman, Sheela; Castelli, Jeffrey P.; Insinga, Frank; Lazauskas, Richard; Furlow, M.C.; Do, Hung; Lockhart, David J.; Wustman, Brandon A.

doi:10.1016/j.nmd.2008.06.266

Neuromuscular Disorders

2008

DOI: 10.1016/j.nmd.2008.06.266

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G.P.11.06 Pharmacological chaperone therapy for Pompe disease: Phase 1 clinical trials and ex vivo response study results

C.W. Pine¹,

Hadis Nafar²,

David J. Palling³

et al.

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G.P.11.06 Pharmacological chaperone therapy for Pompe disease: Phase 1 clinical trials and ex vivo response study results

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