Pharmacological Chaperone Therapy for the Treatment of Gaucher Disease: Results of Phase 1 Clinical Trials and a Clinical Ex Vivo Response Study with a Survey of Blood Markers for... by Weinreb, Neal J., Grabowski, G.A., Pastores, G.M., Fernhoff, P.M., Kaplan, P.B., Palling, D., Pine, C.W., Raines, B.E., Insinga, F., Lockhart, D.J., Do, H.V., Wustman, B.A.

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