Tomo Sawada

Long-term efficacy of hematopoietic stem cell transplantation on brain involvement in patients with mucopolysaccharidosis type II: A nationwide survey in Japan

Akemi Tanaka, Torayuki Okuyama, Yasuyuki Suzuki et al. · Molecular Genetics and Metabolism · 2012 · 147 citations

Medicine, Hematology, Neurology +7

Safety and efficacy of cipaglucosidase alfa plus miglustat versus alglucosidase alfa plus placebo in late-onset Pompe disease (PROPEL): an international, randomised, double-blind, parallel-group, phase 3 trial

Benedikt Schoser, Mark Roberts, Barry J. Byrne et al. · The Lancet Neurology · 2021 · 132 citations · Full text

Pharmacy, Internal Medicine, Pharmacotherapy +3

Acid Ceramidase Deficiency in Mice Results in a Broad Range of Central Nervous System Abnormalities

Jakub Sikora, Shaalee Dworski, E. Ellen Jones et al. · American Journal Of Pathology · 2017 · 49 citations · Full text

Life Sciences, Neurology, Neuroscience +15

Ataxia is the major neuropathological finding in arylsulfatase G-deficient mice: similarities and dissimilarities to Sanfilippo disease (mucopolysaccharidosis type III)

Björn Kowalewski, Peter Heimann, Theresa Ortkras et al. · Human Molecular Genetics · 2014 · 32 citations · Full text

Medicine, Neurology, Pathology +17

104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)

Benedikt Schoser, Priya S. Kishnani, Drago Bratkovic et al. · Journal of Neurology · 2024 · 27 citations · Full text

Pharmacy, Pharmacology, Pharmacotherapy +13