n -乙酰-的疗效和安全性l在儿童和成人亮氨酸GM2 Gangliosidoses

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。

的小儿神经科(K.M.半岛投注体育官网,安德烈亚斯•哈恩), University Children's Hospital (UKGM) and Medical Faculty, Justus Liebig University of Giessen, Giessen, Germany; Department of Pediatrics (K.M.), Medical Faculty and University Hospital, University of Cologne, Cologne, Germa; Department of Neurology (J.C.), Essen University Hospital, University of Duisburg-Essen, Germany; Department of Neurocritical Care, Neurological and Neurosurgical First Stage Rehabilitation and Weaning, MediClin Klinik Reichshof, Germany; Department of Neurologic Diseases and Neurogenetics (J.G.-B.), Institut D'Investigació Biomèdica de Bellvitge, Barcelona, Spain; Department of Neurology (N.G., Anita Hennig, S.S.), Ludwig Maximilian University of Munich, Germany; Department of Neurology (Anhar Hassan), Mayo Clinic, Rochester, MN, United States; Willink Unit (S.J.), Manchester Centre for Genomic Medicine, Royal Manchester Children's Hospital, University of Manchester, United Kingdom; RK Statistics, Brook House, Mesne Lane, Bakewell DE45 1AL, United Kingdom 9. Division of Neurogenetics, New York University Langone, NY, United States; Department of Neurology (H.L.), New York University Langone School of Medicine, NY, United States; Department of Neurology (S.P.), University of California Los Angeles, CA, United States; Department of Adult Metabolic Medicine (R.S.), Salford Royal Foundation NHS Trust, United Kingdom; and Department of Neurology (T.B.-E.), University Hospital Bern (Inselspital),瑞士。