Increasing Enzyme Mannose-6-Phosphate Levels but Not Miglustat Coadministration Enhances the Efficacy of Enzyme Replacement Therapy in Pompe Mice.

Journal

The Journal of pharmacology and experimental therapeutics
ISSN: 1521-0103
Titre abrégé: J Pharmacol Exp Ther
Pays: United States
ID NLM: 0376362

Informations de publication

Date de publication:
Nov 2023
Historique:
received: 03 02 2023
accepted: 01 06 2023
pubmed: 8 9 2023
medline: 8 9 2023
entrez: 7 9 2023
Statut: ppublish

Résumé

Pompe disease is a rare glycogen storage disorder caused by a deficiency in the lysosomal enzyme acid

Identifiants

DOI: 10.1124/jpet.123.001593 PMID: 37679046
pubmed: 37679046
pii: jpet.123.001593
doi: 10.1124/jpet.123.001593
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

188-203

Informations de copyright

Copyright © 2023 by The Author(s).

Auteurs

Allyson Anding (A)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Sofia Kinton (S)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Kaitlyn Baranowski (K)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Alexander Brezzani (A)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Hilde De Busser (H)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Michael R Dufault (MR)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Patrick Finn (P)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Kelly Keefe (K)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Tanya Tetrault (T)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Yi Li (Y)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Weiliang Qiu (W)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Katrien Raes (K)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Olivier Vitse (O)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Mindy Zhang (M)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Robin Ziegler (R)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

S Pablo Sardi (SP)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Bridge Hunter (B)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.).

Kelly George (K)

Metabolic and Lysosomal Storage Disease Research, Rare and Neurologic Diseases Therapeutic Area (A.A., S.K., K.B., A.B., P.F., K.K., T.T., R.Z., S.P.S., B.H., K.G.), Precision Medicine and Computational Biology (M.R.D., M.Z.), and Nonclinical Efficacy and Safety (W.Q.), Sanofi, Cambridge, Massachusetts; Manufacturing Sciences, Analytics, and Technology (MSAT), Sanofi, Geel, Belgium (H.D.B., K.R.); Medicinal Chemistry, Integrated Drug Discovery, Sanofi, Waltham, Massachusetts (Y.L.); and Pharmacokinetics Dynamics and Metabolism, Sanofi, Montpellier, France (O.V.) Kelly.George@sanofi.com.

Classifications MeSH