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Hematopoietic Stem - and Progenitor-Cell Gene Therapy for Hurler Syndrome
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Enzyme Replacement Therapy With Agalsidase Alfa in Patients With Fabrys Disease: An Analysis of Registry Data
Lancet 374:1986-1996, 1950, Mehta,A.,et al, 2009
Rituximab as Treatment for anti-MuSK myasthenia gravis
Neurol 89:1069-1077, Hehir, M.K.,et al, 2017
Long-Term Benefit of Enzyme Replacement Therapy in Pompe Disease
Neurol 89:2365-2373, Kuperus, E.,et al, 2017
Effect of Rituximab in Patients with Leucine-Rich, Glioma-Inactivated 1 Antibody-Associated Encephalopathy
JAMA Neurol 71:896-900, Irani, S.R.,et al, 2014
Long-lasting Treatment Effect of Rituximab in MuSK Myasthenia
Neurol 78:189-193, Diaz-Manera,J.,et al, 2012
Memantine for Dementia in Adults Older than 40 years with Downs Syndrome (MEADOWS): a Randomised,double-blind,placebo-controlled Trial
Lancet 379:528-536, Hanney,M.,et al, 2012
Skin biopsy is useful for the antemortem diagnosis of neuronal intranuclear inclusion disease
Neurol 76:1372-1376, Sone, J.,et al, 2011