부산대학교 도서관

Laugwitz, Lucia ; Schoenmakers, Daphne H. ; Adang, Laura A. ; Beck-Woedl, Stefanie ; Bergner, Caroline ; Bernard, Geneviève ; Bley, Annette ; Boyer, Audrey ; Calbi, Valeria ; Dekker, Hanka ; Eichler, Florian ; Eklund, Erik ; Fumagalli, Francesca ; Gavazzi, Francesco ; Grønborg, Sabine W. ; van Hasselt, Peter ; Langeveld, Mirjam ; Lindemans, Caroline ; Mochel, Fanny ; Oberg, Andreas ; Ram, Dipak ; Saunier-Vivar, Elise ; Schöls, Ludger ; Scholz, Michael ; Sevin, Caroline ; Zerem, Ayelet ; Wolf, Nicole I. ; Groeschel, Samuel

In European Journal of Paediatric Neurology March 2024 49:141-154

Schoenmakers, Daphne H.; Leferink, Prisca S.; Vanderver, Adeline; Bonkowsky, Joshua L.; Kr?eloh-Mann, Ingeborg; Bernard, Genevi?e; Bertini, Enrico; Fatemi, Ali; Fogel, Brent L.; Wolf, Nicole I.; Skwirut, Donna; Buck, Allyson; Holberg, Brett; Saunier-Vivar, Elise F.; Rauner, Robert; Dekker, Hanka; van Bokhoven, Pieter; Stellingwerff, Menno D.; Berkhof, Johannes; van der Knaap, Marjo S.

BMC Neurology. August 17, 2023, Vol. 23 Issue 1

Schoenmakers, Daphne H.; van den Berg, Sibren; Timmers, Lonneke; Adang, Laura A.; Baumer, Tobias; Bosch, Annet; van de Casteele, Marc; Datema, Mareen R.; Dekker, Hanka; Donnelly, Conan; Driessens, Mariette H. E.; Graessner, Holm; Greger, Valerie; Haddad, Tala; Hoglinger, Gunter U.; van den Hout, Hannerieke; Jonker, Carla; Langeveld, Mirjam; Lambert, Laurie J.; Neacy, Eileen; Nieuwland, Marc; Klockgether, Thomas; van der Knaap, Marjo S.; Papadopoulou, Andri; Plueschke, Kelly; van Rijn, Sanne; Rosenberg, Noa; Saunier-Vivar, Elise F.; Vieira, Bruna dos Santos; Hollak, Carla E. M.; Goettsch, Wim G.; Wolf, Nicole I.

NEUROLOGY; SEP 24 2024, 103 6, pe209743 12p.

van der Knaap MS; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Bonkowsky JL; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Vanderver A; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Schiffmann R; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Krägeloh-Mann I; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Bertini E; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Bernard G; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Fatemi SA; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Wolf NI; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Saunier-Vivar E; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Rauner R; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Dekker H; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; van Bokhoven P; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; van de Ven P; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.; Leferink PS; Department of Pediatric Neurology (M.S.v.d.K., N.I.W.), Amsterdam Leukodystrophy Center, Emma Children's Hospital, Amsterdam University Medical Centers; Amsterdam Neuroscience (M.S.v.d.K., N.I.W.); Department of Functional Genomics (M.S.v.d.K.), Center for Neurogenomics and Cognitive Research, Vrije Universiteit, Amsterdam, the Netherlands; Division of Pediatric Neurology (J.L.B.), Department of Pediatrics, University of Utah School of Medicine; Primary Children's Hospital (J.L.B.), Intermountain Healthcare, Salt Lake City, UT; Division of Neurology (A.V.), Children's Hospital of Philadelphia; Department of Neurology (A.V.), Perelman School of Medicine, University of Pennsylvania, PA; 4D Molecular Therapeutics (R.S.), Emeryville, CA; Department of Developmental and Child Neurology (I.K.-M.), Social Pediatrics, University Children's Hospital Tübingen, Germany; Department of Neuroscience (E.B.), Unit of Neuromuscular and Neurodegenerative Diseases, Laboratory of Molecular Medicine, Genetics and Rare Diseases Research Division, IRCCS Ospedale Pediatrico Bambino Gesù, Rome 00146, Italy; Departments of Neurology and Neurosurgery (G.B.), Pediatrics and Human Genetics, McGill University; Department Specialized Medicine (G.B.), Division of Medical Genetics, McGill University Health Center; Child Health and Human Development Program (G.B.), Research Institute of the McGill University Health Center, Montreal, Canada; Kennedy Krieger Institute (S.A.F.), Johns Hopkins University, Baltimore, MD; Research Department (E.S.-V.), European Leukodystrophies Association International and European Leukodystrophies Association France, Paris, France; United Leukodystrophy Foundation (R.R.), DeKalb, IL; Vereniging Volwassenen, Kinderen en Stofwisselingsziekten (H.D.), Zwolle, the Netherlands; Industry Alliance Office (P.v.B., P.S.L.), Amsterdam Neuroscience, Amsterdam University Medical Centers; and Department of Epidemiology and Data Science (P.v.d.V.), Amsterdam University Medical Centers, Vrije Universiteit, Amsterdam, the Netherlands.

Publisher: Published for the American Academy of Neurology by Wolters Kluwer Country of Publication: United States NLM ID: 101671068 Publication Model: eCollection Cited Medium: Print ISSN: 2376-7839 (Print) Linking ISSN: 23767839 NLM ISO Abbreviation: Neurol Genet Subsets: PubMed not MEDLINE