A highly concentrated preparation of sodium 4-phenylbutyrate in an aqueous medium as an alternative for present high dosage therapeutic treatments of certain disorders is provided, specifically for the treatment of spinal muscular atrophy (SMA), central nervous system (CNS) cancer, myelodysplastic syndrome (MS), acute leukemia, glioblastoma multiforme, amyotrophic lateral sclerosis (ALS), and colon cancer.