L-leucine improves anemia and growth in patients with transfusion-dependent Diamond-Blackfan anemia: Results from a multicenter pilot phase I/II study from the Diamond-Blackfan Anemia Registry

Adrianna Vlachos, Evangelia Atsidaftos, Mohammad Lutfi Lababidi, Ellen Muir, Zora R. Rogers, Waseem Alhushki, Jonathan Bernstein, Bertil Glader, Barbara Gruner, Helge Hartung, Christine Knoll, Thomas Loew, Grzegorz Nalepa, Anupama Narla, Arun R. Panigrahi, Colin A. Sieff, Kelly Walkovich, Jason E. Farrar, Jeffrey M. Lipton

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Medicine & Life Sciences