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A validated bowel-preparation tolerability questionnaire and assessment of three commonly used bowel-cleansing agents

A number of approaches targeting deficient GCase activity in GBA-PD have reached the stage of clinical phase 2/3 trials including orally active GCase chaperones to enhance enzymatic activity, substrate reduction therapies modelling current treatment of Gauchers disease and GBA gene therapy (Table 5)

As cells age, mitochondria can become less efficient, leading to a decline in overall cellular function

Vomiting affects one in every 10 patients

Taken together, these results demonstrate that FHA domain residues mediating interaction with P-SF3B1 are essential for efficient pre-mRNA splicing and cell viability