Keywords
> MYOBASE > HEALTH > medicine > disease > disease by system > locomotor system diseases > neuromuscular disease > neuromuscular disease by clinic > motor neuron disease > spinal muscular atrophy > proximal spinal muscular atrophy > SMN1-related proximal spinal muscular atrophy > proximal spinal muscular atrophy type 1
proximal spinal muscular atrophy type 1Synonymsspinal muscular atrophy type 1 ;Werdnig-Hoffman disease spinal muscular type 1 |
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Pechmann A, Author ; Langer T ; Wider S ; Kirschner J | 2018Comment in: Letter to the editor: In reply to Sansone et al. [Eur J Paediatr Neurol. 2018] A 5-center experience with intrathecal administration of nusinersen in SMA1 in Italy letter to the editor of european journal of pediatric neuro[...]Article
Pechmann A, Author ; Langer T ; Schorling D ; Stein S ; Vogt S ; Schara U ; Kolbel H ; Schwartz O ; Hahn A ; Giese K ; Johannsen J ; Denecke J ; Weiss C ; Theophil M ; Kirschner J | 2018Article
Krosschell KJ, Author ; Kissel JT ; Townsend EL ; Simeone SD ; Zhang RZ ; Reyna SP ; Crawford TO ; Schroth MK ; Acsadi G ; Kishnani PS ; von Kleist-Retzow JC ; Hero B ; D'Anjou G ; Smith EC ; Elsheikh B ; Simard LR ; Prior TW ; Scott CB ; LaSalle B ; Sakonju A ; Wirth B ; Swoboda KJ | 2018Article
Ridler C, Author | 01/2018Comment on Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. [N Engl J Med. 2017] Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy. [N Engl J Med. 2017]Article
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De Sanctis R ; Pane M ; Coratti G ; Palermo C ; Leone D ; Pera MC ; Abiusi E ; Fiori S ; Forcina N ; Fanelli L ; Lucibello S ; Mazzone ES ; Tiziano FD ; Mercuri E | 2018Article
Calucho M, Author ; Bernal S ; Alias L ; March F ; Vencesla A ; Rodriguez-Alvarez FJ ; Aller E ; Fernandez RM ; Borrego S ; Millan JM ; Hernandez-Chico C ; Cusco I ; Fuentes-Prior P ; Tizzano EF | 2018Article
Temsah MA, Author ; Al-Sohime FM ; Bashiri FA ; Al-Eyadhy AA ; Hasan GM ; Alhaboob AA | 2018Article
Whittaker HT, Author ; Michell-Robinson MA | 2018Comment in Therapy for Spinal Muscular Atrophy. [N Engl J Med. 2018] Therapy for Spinal Muscular Atrophy. [N Engl J Med. 2018] Comment on Single-Dose Gene-Replacement Therapy for Spinal Muscular Atrophy. [N Engl J Med. 2017] [...]VLM
06/2017L'essai SUNFISH est un essai international de phase 2 mené par la société Hoffman-La Roche.Abstract
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