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Inhibition of Sphingolipid Synthesis as a Phenotype-Modifying Therapy in Cystic Fibrosis

Ben BB, Blacher E, Mantsur E, Schwartz H, Vaknine H, Erez N, et al

CDK4 , a member of the early cell-cycle gene family and not known to be directly involved in response to GH, is the only common gene associated with IGF-I generation in the two cohorts of children

Inclusion of dopaminergic neuronal models (e.g., SH-SY5Y or iPSC-derived midbrain neurons) would provide a more disease-relevant context for evaluating GCLC function

One study found that the sublingual form (which dissolves under the tongue) was more effective than an oral (swallowed) form