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These included amyotrophic lateral sclerosis (ALS), pulmonary fibrosis (PF), acute respiratory distress syndrome (ARDS), Duchenne muscular dystrophy (DMD), cystic fibrosis (CF), Huntingtons disease (HD), pulmonary hypertension (PH), cytokine release syndrome (CRS), frontotemporal dementia (FTD), and graft-versus-host disease (GVHD)., All selected diseases were confirmed to be classified as rare diseases by Orphanet ( a widely used global database for rare diseases

M., Khani, S

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in Handbook of Experimental Pharmacology , 151 (Springer, 2005)

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