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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
3.1.4 Exenatide and its combination with MET: from metabolic improvement to pregnancy outcomes The evidence chain for exenatide is relatively long, encompassing early RCTs focused on menstruation, ovulation, and metabolism, as well as follow-up studies targeting pregnancy outcomes
369 In the phase 1b trial of AMG-232 monotherapy or trametinib in patients with R/R AML, 370 1 patient had DLT, 31% (4/13) of patients expressing wild-type p53 had a response, and no patients expressing mutant p53 had a response
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