2 results
Approved WMOCompleted
Protein arginine methyltransferases (PRMTs) are a subset of enzymes that methylate arginine residues in various cellular proteins including splicing factors, transcription factors, and histone tails. One of these PRMTs, PRMT5, is aberrantly…
Approved WMOWill not start
The purpose of this study is to evaluate the possible risks and efficacy (improvement of disease) with an experimental oral study drug named eliglustat in pediatric patients from 2 to 18 years with Gaucher disease.