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Randomisation versus prioritisation in a managed access programme: Lessons from spinal muscular atrophy.
Servais, Laurent; Kirschner, Janbernd; Muntoni, Francesco
2020In Neuromuscular disorders : NMD, 30 (4), p. 267-269

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Randomisation versus prioritisation in a managed access programme_Lessons from spinal muscular atrophy.pdf

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