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Efficacy and safety of miglustat in the treatment of GM2 gangliosidosis: A systematic review |
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| Authors: | Vahid Mansouri Ali Reza Tavasoli Masoud Khodarahmi Mohammad Sedigh Dakkali Sara Daneshfar Mahmoud Reza Ashrafi Morteza Heidari Sareh Hosseinpour Fariborz Sharifianjazi Maryam Bemanalizadeh |
| Affiliation: | 1. Gene Therapy Research Center, Digestive Diseases Research Institute, Shariati Hospital, Tehran University of Medical Sciences, Tehran, Iran;2. Jefferson Institute of Molecular Medicine, Thomas Jefferson University, Philadelphia, Pennsylvania, USA Pediatric Neurology Division, Department of Pediatrics, Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran;3. Bahar Medical Imaging Center, Karaj, Iran;4. School of Medicine, Zahedan University of Medical Sciences, Zahedan, Iran;5. Faculty of Medicine, Islamic Azad University, Tabriz Branch, Tabriz, Iran;6. Pediatric Neurology Division, Department of Pediatrics, Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran Pediatric Cell and Gene Therapy Research Center (PCGTRC), Tehran University of Medical Sciences, Tehran, Iran;7. Pediatric Neurology Division, Department of Pediatrics, Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran Pediatric Neurology Division, Myelin Disorders Clinic, Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran;8. Division of Pediatric Neurology, Department of Pediatrics, Vali-e-Asr Hospital, Imam Khomeini Hospital Complex, Tehran University of Medical Sciences, Tehran, Iran;9. School of Science and Technology, University of Georgia, Tbilisi, Georgia;10. Pediatric Neurology Division, Department of Pediatrics, Children's Medical Center, Pediatric Center of Excellence, Tehran University of Medical Sciences, Tehran, Iran |
| Abstract: | |
| Keywords: | GM2 gangliosidosis miglustat Sandhoff disease substrate reduction therapy Tay-Sachs disease |