Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.

Autor:	Dimachkie MM; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany. mdimachkie@kumc.edu., Barohn RJ; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Byrne B; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Goker-Alpan O; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Kishnani PS; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Ladha S; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Laforêt P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Mengel KE; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Peña LDM; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Sacconi S; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Straub V; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Trivedi J; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Van Damme P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., van der Ploeg AT; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Vissing J; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Young P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Haack KA; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Foster M; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Gilbert JM; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Miossec P; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Vitse O; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Zhou T; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany., Schoser B; From the University of Kansas Medical Center (M.M.D., R.J.B.), Kansas City; University of Missouri (R.J.B.), Columbia; University of Florida (B.B.), Gainesville; LDRTC (O.G.-A.), Fairfax, VA; Duke University Medical Center (P.S.K., L.D.M.P.), Durham, NC; Barrow Neurological Institute (S.L.), Phoenix, AZ; Centre de Référence des Maladies Neuromusculaires Nord/Est/Ile de France Service de Neurologie (P.L.), Hôpital Raymond-Poincaré, Garches, AP-HP and INSERM U1179, Université Versailles Saint-Quentin-en-Yvelines, Montigny-le-Bretonneux; SphinCS GmbH (K.E.M.), Institute of Clinical Science for LSD, Hochheim, Germany; Cincinnati Children's Hospital Medical Center and University of Cincinnati College of Medicine (L.D.M.P.), OH; Neuromuscular Diseases Centre (S.S.), Department of Clinical Neurosciences, University Hospital of Nice (CHU), France; Newcastle University John Walton Muscular Dystrophy Research Centre (V.S.), Newcastle Hospitals NHS Foundation Trust, United Kingdom; University of Texas Southwestern Medical Center (J.T.), Dallas; Department of Neurosciences (P.V.D.), KU Leuven (Catholic University of Leuven), VIB-Center for Brain & Disease Research, and Department of Neurology, University Hospitals Leuven, Belgium; Erasmus MC University Medical Center (A.T.v.d.P.), Pompe Center & Center for Lysosomal and Metabolic Diseases, Rotterdam, the Netherlands; Copenhagen Neuromuscular Center (J.V.), Rigshospitalet, University of Copenhagen, Denmark; Department of Neurology (P.Y.), Medical Park Bad Feilnbach, Germany; Sanofi (K.A.H., P.M.), Chilly-Mazarin, France; Sanofi (M.F., T.Z.), Cambridge, MA; Elevate Medical Affairs (J.M.G.), Horsham, United Kingdom; Sanofi (O.V.), Montpellier, France; and Friedrich-Baur-Institut (B.S.), Department of Neurology Klinikum München, Germany.
Jazyk:	angličtina
Zdroj:	Neurology [Neurology] 2022 Aug 01; Vol. 99 (5), pp. e536-e548. Date of Electronic Publication: 2022 Aug 01.
DOI:	10.1212/WNL.0000000000200746
Abstrakt:	Background and Objectives: Pompe disease is a rare, progressive neuromuscular disorder caused by deficiency of lysosomal acid α-glucosidase (GAA) and subsequent glycogen accumulation. Avalglucosidase alfa, a recombinant human GAA enzyme replacement therapy designed for increased cellular uptake and glycogen clearance, has been studied for long-term efficacy and safety in patients with late-onset Pompe disease (LOPD). Here, we report up to 6.5 years' experience with avalglucosidase alfa during the NEO1 and NEO-EXT studies. Methods: NEO1 participants with LOPD, either treatment naive (Naive Group) or receiving alglucosidase alfa for ≥9 months (Switch Group), received avalglucosidase alfa (5, 10, or 20 mg/kg every other week [qow]) for 6 months before entering NEO-EXT and continued their NEO1 dose until all proceeded with 20 mg/kg qow. Safety and efficacy, a prespecified exploratory secondary outcome, were assessed; slopes of change for efficacy outcomes were calculated from a repeated mixed-measures model. Results: Twenty-four participants enrolled in NEO1 (Naive Group, n = 10; Switch Group, n = 14); 21 completed and 19 entered NEO-EXT; in February 2020, 17 participants remained in NEO-EXT, with data up to 6.5 years. Avalglucosidase alfa was generally well tolerated during NEO-EXT, with a safety profile consistent with that in NEO1. No deaths or treatment-related life-threatening serious adverse events occurred. Eighteen participants developed antidrug antibodies without apparent effect on clinical outcomes. No participants who were tested developed immunoglobulin E antibodies. Upright forced vital capacity %predicted remained stable in most participants, with slope estimates (95% CIs) of -0.473 per year (-1.188 to 0.242) and -0.648 per year (-1.061 to -0.236) in the Naive and Switch Groups, respectively. Six-minute walk test (6MWT) %predicted was also stable for most participants, with slope estimates of -0.701 per year (-1.571 to 0.169) and -0.846 per year (-1.567 to -0.125) for the Naive and Switch Groups, respectively. Improvements in 6MWT distance were observed in most participants aged <45 years at NEO1 enrollment in both the Naive and Switch Groups. Discussion: Avalglucosidase alfa was generally well tolerated for up to 6.5 years in adult participants with LOPD either naive to alglucosidase alfa or who had previously received alglucosidase alfa for ≥9 months. Classification of Evidence: This study provides Class IV evidence of long-term tolerability and sustained efficacy of avalglucosidase alfa in patients with LOPD after up to 6.5 years. Trial Registration Information: NCT01898364 (NEO1 first posted: July 12, 2013; clinicaltrials.gov/ct2/show/NCT01898364); NCT02032524 (NEO-EXT first posted: January 10, 2014; clinicaltrials.gov/ct2/show/NCT02032524). First participant enrollment: NEO1-August 19, 2013; NEO-EXT-February 27, 2014. (Copyright © 2022 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.)
Databáze:	MEDLINE
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