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1 (+13.8 to +16.4; P less then 0.0001), and the mean (95% confidence interval) in weight was +4.2 kg (+3.9 to +4.6; P less then 0.0001). The number of patients requiring long-term oxygen, noninvasive ventilation, and/or enteral tube feeding decreased by 50%, 30%, and 50%, respectively (P less then 0.01). Although 16 patients were on the transplant waiting list and 37 were undergoing transplantation evaluation at treatment initiation, only 2 received a transplant, and 1 died. By September 2020, only five patients were still on the
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