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Hoffman, E.P.; Bonnemann, C.; Boutin, M.; Brais, B.; Buccella, F.; Burghes, A.; ... ; TREAT-NMD Alliance
2017
Facilitating orphan drug development: Proceedings of the TREAT-NMD International Conference, December 2015, Washington, DC, USA
Article / Letter to editor
metadata only
Bladen, C.L.; Salgado, D.; Monges, S.; Foncuberta, M.E.; Kekou, K.; Kosma, K.; ... ; Lochmuller, H.
2015
The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations
Article / Letter to editor
metadata only
Bladen, C.L.; Thompson, R.; Jackson, J.M.; Garland, C.; Wegel, C.; Ambrosini, A.; ... ; Lochmuller, H.
2014
Mapping the differences in care for 5,000 Spinal Muscular Atrophy patients, a survey of 24 national registries in North America, Australasia and Europe
Article / Letter to editor
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Bladen, C.L.; Rafferty, K.; Straub, V.; Monges, S.; Moresco, A.; Dawkins, H.; ... ; Lochmuller, H.
2013
The TREAT-NMD Duchenne Muscular Dystrophy Registries: Conception, Design, and Utilization by Industry and Academia
Article / Letter to editor
metadata only
