P.149The MYODA operational seamless clinical trial design phase I to III: a new approach for rare diseases to evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of BIO101 (MAS activator) in paediatric patients with a genetically confirmed diagnosis of Duchenne muscular dystrophy

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Bibliographic Details
Published inNeuromuscular disorders : NMD Vol. 29; p. S92
Main Authors Chabane, M., Dioh, W., Dilda, P., Lafont, R., Veillet, S., Voit, T., Agus, S.
Format Journal Article
LanguageEnglish
Published Elsevier B.V 01.10.2019
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ISSN:0960-8966
1873-2364
DOI:10.1016/j.nmd.2019.06.205