DOI:
10.1055/s-00000041
Neuropediatrics
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References
Heslop E, Csimma C, Straub V. , et al; TACT.
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development.
Orphanet J Rare Dis 2015;
10: 49
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